Trial Outcomes & Findings for Thymus Transplantation Dose in DiGeorge #932 (NCT NCT00576836)
NCT ID: NCT00576836
Last Updated: 2022-03-25
Results Overview
Survival at 1 year post CTTI was assessed using the Kaplan Meier Estimated Survival. This mathematical function estimates the survival for a certain length of time.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
7 participants
Primary outcome timeframe
1 year post-CTTI
Results posted on
2022-03-25
Participant Flow
The goal of this study is survival of subjects after cultured thymus tissue implantation (CTTI) regardless of parathyroid co-transplantation. No subject underwent CTTI with parathyroid transplant.
Participant milestones
| Measure |
Cultured Thymus Tissue Implantation w Parathyroid Transplant
Cultured Thymus Tissue Implantation (CTTI): Thymus tissue (from unrelated donor), thymus donor and thymus donor's birth mother screened for safety. CTTI done under general anesthesia. Cultured thymus tissue is implanted into quadriceps. Thymus dose at least 4grams/m2 body surface area (0.2 grams/kg body weight) and not \>18 grams/m2 body surface area (1.0 grams/kg body weight). At time of CTTI, skin biopsy is obtained to look for preexisting T cells. 2-3 months post-CTTI allograft biopsy done to evaluate for thymopoiesis \& graft rejection. At time of biopsy, skin biopsy done to look for T cell clonal populations. Allograft biopsy not done if subject medically unstable. Post-CTTI, subjects followed by immune evaluations, using blood samples.
Parathyroid Tissue for Transplantation: If both parents meet eligibility criteria, the parent chosen for donation will be the one sharing the parental HLA (Human Leukocyte Antigen)-DR allele that is not in the recipient but is in the thymus donor.
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Cultured Thymus Tissue Implantation
Cultured Thymus Tissue Implantation (CTTI): Thymus tissue (from unrelated donor), thymus donor, and thymus donor's birth mother screened for safety. CTTI is done under general anesthesia. Cultured thymus tissue is implanted into quadriceps. Cultured thymus tissue dose at least 4grams/m2 body surface area (0.2 grams/kg body weight) and not \>18 grams/m2 body surface area (1.0 grams/kg body weight). At time of CTTI, skin biopsy obtained to look for preexisting T cells. 2-3 months post-CTTI allograft biopsy done to evaluate for thymopoiesis \& graft rejection. At time of biopsy, skin biopsy is done to look for T cell clonal populations. (Allograft biopsy not done if subject medically unstable.) Post-CTTI, subjects followed by immune evaluations, using blood samples.
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|---|---|---|
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Overall Study
STARTED
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0
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7
|
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Overall Study
COMPLETED
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0
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5
|
|
Overall Study
NOT COMPLETED
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0
|
2
|
Reasons for withdrawal
| Measure |
Cultured Thymus Tissue Implantation w Parathyroid Transplant
Cultured Thymus Tissue Implantation (CTTI): Thymus tissue (from unrelated donor), thymus donor and thymus donor's birth mother screened for safety. CTTI done under general anesthesia. Cultured thymus tissue is implanted into quadriceps. Thymus dose at least 4grams/m2 body surface area (0.2 grams/kg body weight) and not \>18 grams/m2 body surface area (1.0 grams/kg body weight). At time of CTTI, skin biopsy is obtained to look for preexisting T cells. 2-3 months post-CTTI allograft biopsy done to evaluate for thymopoiesis \& graft rejection. At time of biopsy, skin biopsy done to look for T cell clonal populations. Allograft biopsy not done if subject medically unstable. Post-CTTI, subjects followed by immune evaluations, using blood samples.
Parathyroid Tissue for Transplantation: If both parents meet eligibility criteria, the parent chosen for donation will be the one sharing the parental HLA (Human Leukocyte Antigen)-DR allele that is not in the recipient but is in the thymus donor.
|
Cultured Thymus Tissue Implantation
Cultured Thymus Tissue Implantation (CTTI): Thymus tissue (from unrelated donor), thymus donor, and thymus donor's birth mother screened for safety. CTTI is done under general anesthesia. Cultured thymus tissue is implanted into quadriceps. Cultured thymus tissue dose at least 4grams/m2 body surface area (0.2 grams/kg body weight) and not \>18 grams/m2 body surface area (1.0 grams/kg body weight). At time of CTTI, skin biopsy obtained to look for preexisting T cells. 2-3 months post-CTTI allograft biopsy done to evaluate for thymopoiesis \& graft rejection. At time of biopsy, skin biopsy is done to look for T cell clonal populations. (Allograft biopsy not done if subject medically unstable.) Post-CTTI, subjects followed by immune evaluations, using blood samples.
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|---|---|---|
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Overall Study
Death
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0
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1
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Overall Study
Physician Decision
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0
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1
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Baseline Characteristics
Thymus Transplantation Dose in DiGeorge #932
Baseline characteristics by cohort
| Measure |
Cultured Thymus Tissue Implantation
n=7 Participants
Cultured Thymus Tissue Implantation (CTTI) (previously described as transplantation) is done using allogenic cultured postnatal tissue from unrelated donors.
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|---|---|
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Age, Categorical
<=18 years
|
7 Participants
n=93 Participants
|
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Age, Categorical
Between 18 and 65 years
|
0 Participants
n=93 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
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Age, Continuous
|
316 days
STANDARD_DEVIATION 182 • n=93 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=93 Participants
|
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Sex: Female, Male
Male
|
4 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
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2 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
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Race (NIH/OMB)
Black or African American
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1 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
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Region of Enrollment
United States
|
7 participants
n=93 Participants
|
PRIMARY outcome
Timeframe: 1 year post-CTTIPopulation: Analysis includes cDGA participants. After CTTI, 1 subject was determined to have SCID and not cDGA. Efficacy analysis as reported is on cDGA, without the SCID subject as CTTI cannot lead to T cell development in SCID. No subjects were enrolled into Arm 1. No subjects received parathyroid transplant. Therefore, results are reported for Arm 2 only.
Survival at 1 year post CTTI was assessed using the Kaplan Meier Estimated Survival. This mathematical function estimates the survival for a certain length of time.
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=6 Participants
Cultured Thymus Tissue Implantation (CTTI) (previously described as transplantation) is done using allogenic cultured postnatal tissue from unrelated donors.
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|---|---|
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Survival at 1 Year Post-CTTI
|
83 % of participants who survive to 1 year
Interval 27.3 to 97.5
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SECONDARY outcome
Timeframe: 2 years post-CTTIPopulation: Analysis includes cDGA participants. After CTTI, 1 subject was determined to have SCID and not cDGA. Efficacy analysis as reported is on cDGA, without the SCID subject as CTTI cannot lead to T cell development in SCID. No subjects were enrolled into Arm 1. No subjects received parathyroid transplant. Therefore, results are reported for Arm 2 only.
Survival at 2 years post CTTI was assessed using the Kaplan Meier Estimated Survival. This mathematical function estimates the survival for a certain length of time.
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=6 Participants
Cultured Thymus Tissue Implantation (CTTI) (previously described as transplantation) is done using allogenic cultured postnatal tissue from unrelated donors.
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|---|---|
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Survival at 2 Years Post-CTTI
|
83 % of participants who survive to 2 years
Interval 27.3 to 97.5
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SECONDARY outcome
Timeframe: 1 year post-CTTIPopulation: Data were only included on cDGA participants for the 1 year time point if a CD3 T cell count was performed in the relevant time period. The study was designed to assess survival. No subjects were enrolled into Arm 1. No subjects received a parathyroid transplant. Therefore, results are reported for Arm 2 only.
The development of total CD3 T cells at one year as measured using flow cytometry
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=4 Participants
Cultured Thymus Tissue Implantation (CTTI) (previously described as transplantation) is done using allogenic cultured postnatal tissue from unrelated donors.
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|---|---|
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Immune Reconstitution Efficacy - CD3 T Cells
|
635 cells/mm3
Interval 268.0 to 1400.0
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SECONDARY outcome
Timeframe: 1 year post-CTTIPopulation: Data were only included on cDGA participants for the 1 year time point if a CD4 T cell count was performed in the relevant time period. The study was designed to assess survival. No subjects were enrolled into Arm 1. No subjects received a parathyroid transplant. Therefore, results are reported for Arm 2 only.
The development of total CD4 T cells at one year as measured using flow cytometry
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=4 Participants
Cultured Thymus Tissue Implantation (CTTI) (previously described as transplantation) is done using allogenic cultured postnatal tissue from unrelated donors.
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|---|---|
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Immune Reconstitution Efficacy - CD4 T Cells
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499 cells/mm3
Interval 224.0 to 760.0
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SECONDARY outcome
Timeframe: 1 year post-CTTIPopulation: Data were only included on cDGA participants for the 1 year time point if a CD8 T cell count was performed in the relevant time period. The study was designed to assess survival. No subjects were enrolled into Arm 1. No subjects received a parathyroid transplant. Therefore, results are reported for Arm 2 only.
The development of total CD8 T cells at one year as measured using flow cytometry
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=4 Participants
Cultured Thymus Tissue Implantation (CTTI) (previously described as transplantation) is done using allogenic cultured postnatal tissue from unrelated donors.
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|---|---|
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Immune Reconstitution Efficacy - CD8 T Cells
|
116 cells/mm3
Interval 47.0 to 394.0
|
SECONDARY outcome
Timeframe: 1 year post-CTTIPopulation: Data were only included on cDGA participants for the 1 year time point if a T cell count was performed in the relevant time period. The study was designed to assess survival. No subjects were enrolled into Arm 1. No subjects received a parathyroid transplant. Therefore, results are reported for Arm 2 only.
The development of naive CD4 T cells at one year as measured using flow cytometry
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=4 Participants
Cultured Thymus Tissue Implantation (CTTI) (previously described as transplantation) is done using allogenic cultured postnatal tissue from unrelated donors.
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|---|---|
|
Immune Reconstitution Efficacy - Naive CD4 T Cells
|
279 cells/mm3
Interval 97.0 to 476.0
|
SECONDARY outcome
Timeframe: 1 year post-CTTIPopulation: Data were only included on cDGA participants for the 1 year time point if a T cell count was performed in the relevant time period. The study was designed to assess survival. No subjects were enrolled into Arm 1. No subjects received a parathyroid transplant. Therefore, results are reported for Arm 2 only.
The development of naïve CD8 T cells at one year as measured using flow cytometry.
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=2 Participants
Cultured Thymus Tissue Implantation (CTTI) (previously described as transplantation) is done using allogenic cultured postnatal tissue from unrelated donors.
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|---|---|
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Immune Reconstitution Efficacy - Naive CD8 T Cells
|
214 cells/mm3
Interval 124.0 to 304.0
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SECONDARY outcome
Timeframe: 1 year post-CTTIPopulation: Data were only included on cDGA participants for the 1 year time point if testing was performed in the relevant time period. The study was designed to assess survival. No subjects were enrolled into Arm 1. No subjects received a parathyroid transplant. Therefore, results are reported for Arm 2 only.
The development of a T cell proliferative response to the mitogen phytohemagglutinin.
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=4 Participants
Cultured Thymus Tissue Implantation (CTTI) (previously described as transplantation) is done using allogenic cultured postnatal tissue from unrelated donors.
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|---|---|
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Immune Reconstitution Efficacy - Response to Mitogens
|
135016 counts per minute (cpm)
Interval 84404.0 to 190928.0
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SECONDARY outcome
Timeframe: 2 to 3 months post-CTTIPopulation: cDGA participants who had a biopsy on the thymus tissue implanted.The study was designed to assess survival. No subjects were enrolled into Arm 1. No subjects received a parathyroid transplant. Therefore, results are reported for Arm 2 only.
Evidence, on biopsy of the thymus tissue implanted in the recipient muscle, that shows the development of new T cells.
Outcome measures
| Measure |
Cultured Thymus Tissue Implantation
n=2 Participants
Cultured Thymus Tissue Implantation (CTTI) (previously described as transplantation) is done using allogenic cultured postnatal tissue from unrelated donors.
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|---|---|
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Thymus Allograft Biopsy
Evidence of thymopoiesis
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1 Participants
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Thymus Allograft Biopsy
Evidence of rejection
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0 Participants
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Thymus Allograft Biopsy
Inconclusive for thymopoiesis
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1 Participants
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Adverse Events
Cultured Thymus Tissue Implantation
Serious events: 6 serious events
Other events: 7 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Cultured Thymus Tissue Implantation
n=7 participants at risk
Cultured Thymus Tissue Implantation (CTTI) (previously described as transplantation) is done using allogenic cultured postnatal tissue from unrelated donors.
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|---|---|
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Infections and infestations
Device related infection
|
57.1%
4/7 • Number of events 12 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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General disorders
Pyrexia
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42.9%
3/7 • Number of events 5 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Respiratory, thoracic and mediastinal disorders
Hypoxia
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42.9%
3/7 • Number of events 3 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Infections and infestations
Pneumonia
|
28.6%
2/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
28.6%
2/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Immune system disorders
Hypersensitivity
|
14.3%
1/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Infections and infestations
Respiratory tract infection bacterial
|
14.3%
1/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Renal and urinary disorders
Renal failure
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14.3%
1/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Blood and lymphatic system disorders
Autoimmune haemolytic anaemia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Blood and lymphatic system disorders
Haemolysis
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Blood and lymphatic system disorders
Neutropenia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Blood and lymphatic system disorders
Thrombocytopenia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Congenital, familial and genetic disorders
Tracheo-oesophageal fistula
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Gastrointestinal disorders
Abdominal distension
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Gastrointestinal disorders
Diarrhoea
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Gastrointestinal disorders
Gastrointestinal haemorrhage
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
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Immune system disorders
Graft versus host disease
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
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Immune system disorders
Graft versus host disease in gastrointestinal tract
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Adenoviral upper respiratory infection
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Brain abscess
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Enterobacter bacteraemia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Enterococcal bacteraemia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Liver abscess
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Lower respiratory tract infection
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Lower respiratory tract infection bacterial
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Otitis media
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Pneumonia klebsiella
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Pneumonia pseudomonal
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Injury, poisoning and procedural complications
Transfusion reaction
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Investigations
Lymphocyte count abnormal
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Metabolism and nutrition disorders
Electrolyte imbalance
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Nervous system disorders
Central nervous system haemorrhage
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Vascular disorders
Hypotension
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
Other adverse events
| Measure |
Cultured Thymus Tissue Implantation
n=7 participants at risk
Cultured Thymus Tissue Implantation (CTTI) (previously described as transplantation) is done using allogenic cultured postnatal tissue from unrelated donors.
|
|---|---|
|
General disorders
Pyrexia
|
57.1%
4/7 • Number of events 10 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Blood and lymphatic system disorders
Anaemia
|
42.9%
3/7 • Number of events 4 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Investigations
Alanine aminotransferase increased
|
42.9%
3/7 • Number of events 3 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Investigations
Aspartate aminotransferase increased
|
42.9%
3/7 • Number of events 3 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Cardiac disorders
Sinus tachycardia
|
28.6%
2/7 • Number of events 3 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Pneumonia pseudomonal
|
28.6%
2/7 • Number of events 3 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Metabolism and nutrition disorders
Feeding intolerance
|
28.6%
2/7 • Number of events 3 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
28.6%
2/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
28.6%
2/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Urinary tract infection enterococcal
|
28.6%
2/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Investigations
Blood bilirubin increased
|
28.6%
2/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Investigations
International normalised ratio increased
|
28.6%
2/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Nervous system disorders
Seizure
|
28.6%
2/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Nervous system disorders
Tremor
|
28.6%
2/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
28.6%
2/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Vascular disorders
Hypertension
|
28.6%
2/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Lower respiratory tract infection bacterial
|
14.3%
1/7 • Number of events 4 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Gastrointestinal disorders
Diarrhoea
|
14.3%
1/7 • Number of events 2 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Cardiac disorders
Ventricular extrasystoles
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Cardiac disorders
Ventricular tachycardia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Ear and labyrinth disorders
Deafness
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Ear and labyrinth disorders
Middle ear effusion
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Endocrine disorders
Cushingoid
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Eye disorders
Retinal haemorrhage
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Gastrointestinal disorders
Abdominal distension
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Gastrointestinal disorders
Retching
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Gastrointestinal disorders
Vomiting
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
General disorders
Catheter site erosion
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Hepatobiliary disorders
Cholestasis
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Hepatobiliary disorders
Hepatic lesion
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Hepatobiliary disorders
Hepatitis
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Clostridium difficile colitis
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Cystitis escherichia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Ear infection
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Enterobacter pneumonia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Oropharyngeal candidiasis
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Parainfluenzae virus infection
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Pneumocystis jirovecii pneumonia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Sinusitis bacterial
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Stoma site infection
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Upper respiratory tract infection bacterial
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Urinary tract infection bacterial
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Urinary tract infection fungal
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Urinary tract infection pseudomonal
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Viraemia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Injury, poisoning and procedural complications
Wound complication
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Investigations
Activated partial thromboplastin time prolonged
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Investigations
Amylase increased
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Investigations
Blood alkaline phosphatase increased
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Investigations
Blood bicarbonate decreased
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Investigations
Blood urea increased
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Investigations
Urine electrolytes abnormal
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Investigations
pH urine increased
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Metabolism and nutrition disorders
Hypernatraemia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Metabolism and nutrition disorders
Hypertriglyceridaemia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Metabolism and nutrition disorders
Lactase deficiency
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Musculoskeletal and connective tissue disorders
Osteoporosis
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Nervous system disorders
Peripheral motor neuropathy
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Nervous system disorders
Vocal cord paralysis
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Skin and subcutaneous tissue disorders
Drug eruption
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Skin and subcutaneous tissue disorders
Skin disorder
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
|
Vascular disorders
Hypotension
|
14.3%
1/7 • Number of events 1 • Two years post-CTTI
AE reporting on all participants (cDGA and SCID) who received cultured thymus tissue Implantation (CTTI) (previously described as transplantation). No participants received CTTI with a Parathyroid Transplant.
|
Additional Information
M. Louise Markert, MD, PhD Professor of Pediatrics and Immunology
Duke University Medical Center
Phone: 919-684-6263
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place