Trial Outcomes & Findings for Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease (NCT NCT01565616)
NCT ID: NCT01565616
Last Updated: 2017-11-21
Results Overview
Event-free survival is defined as stable donor erythropoiesis with no new clinical evidence of sickle cell disease. Primary or late graft rejection, disease recurrence, and death are considered events for this endpoint.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
22 participants
Primary outcome timeframe
1 year after transplant
Results posted on
2017-11-21
Participant Flow
Participants were enrolled from 8 study locations between March, 2012 and June, 2015.
The first component of the study was restricted to 5 patients with a related donor. If no more than 2 of the first 5 patients experienced unacceptable toxicity within six months of transplantation then the safety of the regimen was considered promising and the study could include patients with a related or unrelated HLA matched donor.
Participant milestones
| Measure |
Bone Marrow Transplant Recipients
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
|
|---|---|
|
Overall Study
STARTED
|
22
|
|
Overall Study
First Component: 5 Related Donors
|
5
|
|
Overall Study
Second Component: Related or Unrelated
|
17
|
|
Overall Study
COMPLETED
|
22
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease
Baseline characteristics by cohort
| Measure |
Bone Marrow Transplant Recipients
n=22 Participants
Individuals receiving a bone marrow transplant at one of 10 study locations, between March 2012 and June, 2015.
|
|---|---|
|
Age, Customized
16-19 years of age
|
6 Participants
n=5 Participants
|
|
Age, Customized
20-24 years of age
|
8 Participants
n=5 Participants
|
|
Age, Customized
24-29 years of age
|
6 Participants
n=5 Participants
|
|
Age, Customized
30-40 years of age
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
22 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
22 Participants
n=5 Participants
|
|
Donor Relation
Related
|
17 Participants
n=5 Participants
|
|
Donor Relation
Unrelated
|
5 Participants
n=5 Participants
|
|
Indication of Severe Sickle Cell Disease
Significant neurological event (stroke)
|
2 Participants
n=5 Participants
|
|
Indication of Severe Sickle Cell Disease
Acute chest syndrome
|
2 Participants
n=5 Participants
|
|
Indication of Severe Sickle Cell Disease
Recurrent pain events
|
15 Participants
n=5 Participants
|
|
Indication of Severe Sickle Cell Disease
Regular RBC transfusion therapy
|
4 Participants
n=5 Participants
|
|
Indication of Severe Sickle Cell Disease
Elevated TRJ Velocity
|
4 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 year after transplantEvent-free survival is defined as stable donor erythropoiesis with no new clinical evidence of sickle cell disease. Primary or late graft rejection, disease recurrence, and death are considered events for this endpoint.
Outcome measures
| Measure |
Bone Marrow Transplant Recipients
n=22 Participants
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
|
|---|---|
|
Event -Free Survival Rate
|
19 Participants
|
SECONDARY outcome
Timeframe: 1 year after transplantPrimary graft failure occurs when a transplant recipient does not achieve donor chimerism following a bone marrow transplant. Secondary graft failure occurs when graft fails after donor chimerism had initially occurred.
Outcome measures
| Measure |
Bone Marrow Transplant Recipients
n=22 Participants
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
|
|---|---|
|
Graft Failure
Primary graft failure
|
0 Participants
|
|
Graft Failure
Secondary graft failure
|
1 Participants
|
SECONDARY outcome
Timeframe: 1 year after transplantAcute GVHD was graded according to the Center for International Blood and Marrow Transplant Research (CIBMTR) consensus criteria. Clinical manifestations of acute GVHD include skin, liver, and gastrointestinal symptoms. Grading of acute GVHD is determined by size of maculopapular rash, bilirubin and stool output. Acute GVHD grades range from 0 to 4 with 0 indicating no GVHD and 4 representing the most severe grade. Grade II is defined as a maculopapular rash over 25-50% of body surface area (BSA), bilirubin of 3.1 to 6 mg/dL, and stool output of 1000-1500 mL/d (for adults). Grade III is defined as a maculopapular rash over more than 50% of BSA, bilirubin of 6.1 to 15 mg/dL, and stool output of greater than 1500 mL/d (for adults). Grade IV is defined as generalized erythroderma with bullous formation, bilirubin greater than 15 mg/dL, and severe abdominal pain with or without ileus.
Outcome measures
| Measure |
Bone Marrow Transplant Recipients
n=22 Participants
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
|
|---|---|
|
Acute Graft Versus Host Disease (GVHD)
Grade II Acute GVHD
|
3 Participants
|
|
Acute Graft Versus Host Disease (GVHD)
Grade III Acute GVHD
|
1 Participants
|
|
Acute Graft Versus Host Disease (GVHD)
Grade IV Acute GVHD
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 year after transplantChronic GVHD was graded according to the National Institutes of Health (NIH) 2014 Consensus Criteria Diagnosis and scoring the severity of chronic GVHD is determined by evaluating symptoms of the skin, nails, hair, mouth, eyes, genitalia, gastrointestinal tract, liver, lungs, muscles, fascia and joints, immune function as well as other symptoms such as ascites and neuropathy. Chronic GVHD is graded as mild, moderate or severe based on the number of organ sites impacted and the severity of symptoms.
Outcome measures
| Measure |
Bone Marrow Transplant Recipients
n=22 Participants
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
|
|---|---|
|
Chronic Graft Versus Host Disease (GVHD)
Mild Chronic GVHD
|
3 Participants
|
|
Chronic Graft Versus Host Disease (GVHD)
Moderate Chronic GVHD
|
2 Participants
|
|
Chronic Graft Versus Host Disease (GVHD)
Severe Chronic GVHD
|
1 Participants
|
SECONDARY outcome
Timeframe: 1 year after transplantOverall survival is defined as survival with or without sickle cell disease after hematopoietic cell transplantation (HCT).
Outcome measures
| Measure |
Bone Marrow Transplant Recipients
n=22 Participants
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
|
|---|---|
|
Overall Survival
|
20 Participants
|
SECONDARY outcome
Timeframe: 1 year after transplantTime to neutrophil engraftment is defined as the first of 3 measurements on different days when the patient has an absolute neutrophil count of at least 500/µL after conditioning. Time to Platelet engraftment is defined as the first day of a minimum of 3 measurements on different days that the patient has achieved a platelet count \> 50,000/µL, without receiving a platelet transfusion in the previous 7 days.
Outcome measures
| Measure |
Bone Marrow Transplant Recipients
n=22 Participants
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
|
|---|---|
|
Time to Neutrophil and Platelet Engraftment
Neutrophil engraftment
|
17 Days
Interval 12.0 to 26.0
|
|
Time to Neutrophil and Platelet Engraftment
Platelet
|
21 Days
Interval 13.0 to 26.0
|
SECONDARY outcome
Timeframe: 1 year after transplantCommon transplant related complications were monitored as a secondary outcome measure of this study. These transplant related complications include hepatic veno-occlusive disease (VOD), idiopathic pneumonia syndrome (IPS), central nervous system (CNS) toxicity complications of posterior reversible encephalopathy syndrome (PRES), hemorrhage, and seizures, cytomegalovirus (CMV) infection, adenovirus infection, Epstein-Barr virus (EBV) infection, post-transplant lymphoproliferative disease (PTLD), and invasive fungal infection.
Outcome measures
| Measure |
Bone Marrow Transplant Recipients
n=22 Participants
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
|
|---|---|
|
Transplant Related Outcomes
Hepatic Veno-occlusive Disease (VOD)
|
0 Participants
|
|
Transplant Related Outcomes
Idiopathic Pneumonia Syndrome (IPS)
|
1 Participants
|
|
Transplant Related Outcomes
PRES
|
1 Participants
|
|
Transplant Related Outcomes
Central Nervous System Toxicity: Hemorrhage
|
1 Participants
|
|
Transplant Related Outcomes
Central Nervous System Toxicity: Seizure
|
1 Participants
|
|
Transplant Related Outcomes
Cytomegalovirus (CMV) Infection
|
11 Participants
|
|
Transplant Related Outcomes
Adenovirus Infection
|
0 Participants
|
|
Transplant Related Outcomes
Epstein-Barr Virus (EBV) Infection
|
4 Participants
|
|
Transplant Related Outcomes
Post-transplant Lymphoproliferative Disease
|
1 Participants
|
|
Transplant Related Outcomes
Invasive Fungal Infection
|
0 Participants
|
POST_HOC outcome
Timeframe: Baseline, 1 year after transplantPopulation: This outcome measure compared quality of life scores one year post-HCT to baseline in the 17 participants who completed the surveys.
Health related quality of life was measured with the 57 item Patient-Reported Outcomes Measurement Information System (PROMIS-57). The PROMIS-57 includes 8 domains of health. The domains of Anxiety, Depression, Fatigue, Pain Interference, Physical Function, Satisfaction with Social Role, and Sleep Disturbances each include 8 items. Respondents indicate the degree to which statements about specific health issues are problematic on a scale of 1 to 5 and responses are converted to a t-score metric. A score of 50 is the mean score for the general population in the United States, with a standard deviation of 10. Scores above 50 indicate the topic of the domain is being experienced more than average while scores below 50 mean that it is less than average. The domain of Pain Intensity is measured with a single item asking participants to rate their average pain on a scale from 0 (no pain) to 10 (worst imaginable pain). The raw mean score is used.
Outcome measures
| Measure |
Bone Marrow Transplant Recipients
n=17 Participants
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
|
|---|---|
|
PROMIS-57 Scores Health Related Quality of Life
Anxiety domain score: baseline
|
50.6 units on a scale
Standard Deviation 10.4
|
|
PROMIS-57 Scores Health Related Quality of Life
Anxiety domain score: 1 year
|
48.1 units on a scale
Standard Deviation 10.5
|
|
PROMIS-57 Scores Health Related Quality of Life
Depression domain score: baseline
|
44.7 units on a scale
Standard Deviation 6.7
|
|
PROMIS-57 Scores Health Related Quality of Life
Depresssion domain score: 1 year
|
46.9 units on a scale
Standard Deviation 10.3
|
|
PROMIS-57 Scores Health Related Quality of Life
Fatigue domain score: baseline
|
48.0 units on a scale
Standard Deviation 10.5
|
|
PROMIS-57 Scores Health Related Quality of Life
Fatigue domain score: 1 year
|
46.1 units on a scale
Standard Deviation 13.4
|
|
PROMIS-57 Scores Health Related Quality of Life
Pain interference domain score: baseline
|
58.5 units on a scale
Standard Deviation 10.3
|
|
PROMIS-57 Scores Health Related Quality of Life
Pain interference domain score: 1 year
|
50.1 units on a scale
Standard Deviation 11.6
|
|
PROMIS-57 Scores Health Related Quality of Life
Physical function domain score: baseline
|
44.1 units on a scale
Standard Deviation 8.0
|
|
PROMIS-57 Scores Health Related Quality of Life
Physical function domain score: 1 year
|
50.1 units on a scale
Standard Deviation 11.0
|
|
PROMIS-57 Scores Health Related Quality of Life
Social role satisfaction domain score: baseline
|
51.7 units on a scale
Standard Deviation 11.6
|
|
PROMIS-57 Scores Health Related Quality of Life
Social role satisfaction domain score: 1 year
|
52.4 units on a scale
Standard Deviation 12.3
|
|
PROMIS-57 Scores Health Related Quality of Life
Sleep disturbance domain score: baseline
|
47.7 units on a scale
Standard Deviation 11.9
|
|
PROMIS-57 Scores Health Related Quality of Life
Sleep disturbance domain score: 1 year
|
46.9 units on a scale
Standard Deviation 9.6
|
|
PROMIS-57 Scores Health Related Quality of Life
Pain intensity domain score: baseline
|
3.2 units on a scale
Standard Deviation 3.4
|
|
PROMIS-57 Scores Health Related Quality of Life
Pain intensity domain score: 1 year
|
2.4 units on a scale
Standard Deviation 3.0
|
Adverse Events
Bone Marrow Transplant Recipients
Serious events: 14 serious events
Other events: 12 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Bone Marrow Transplant Recipients
n=22 participants at risk
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
|
|---|---|
|
Gastrointestinal disorders
Hospitalization due to abdominal pain, diarrhea, and bloody stools
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Gastrointestinal disorders
Hospitalization due to abdominal pain, nausea, vomiting, and elevated liver enzymes
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Respiratory, thoracic and mediastinal disorders
Hospitalization due to acute inflammatory bronchitis
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Hospitalization due to acute on chronic pain
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Blood and lymphatic system disorders
Anemia secondary to hemolysis
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Respiratory, thoracic and mediastinal disorders
Hospitalization due to chest pain, secondary to pneumonia
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Hepatobiliary disorders
Cholelithiasis
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Hepatobiliary disorders
Elevated liver enzymes
|
9.1%
2/22 • Number of events 3 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Surgical and medical procedures
Hospitalization due to elevated prograf level
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Hospitalization due to exacerbation of pain syndrome
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Facial swelling with sore throat
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Infections and infestations
Hospitalization due to fever, malaise, and chills
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Hepatobiliary disorders
Gallbladder obstruction
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Hepatobiliary disorders
Gallstones with hyperbilirubinemia
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Gastrointestinal disorders
Gastritis
|
4.5%
1/22 • Number of events 2 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Generalized pain
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Headache requiring Norco
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Blood and lymphatic system disorders
Hemolysis with rising creatinine
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Blood and lymphatic system disorders
Hypercalcemia
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Gastrointestinal disorders
Mucositis
|
9.1%
2/22 • Number of events 2 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Mucosistis, esophageal pain, and low back pain
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Nervous system disorders
Neurological changes
|
9.1%
2/22 • Number of events 2 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Injury, poisoning and procedural complications
Opioid-induced over-sedation
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Blood and lymphatic system disorders
Hospitalization due to pain crisis
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Cardiac disorders
Hospitalization due to pericardial effusion
|
9.1%
2/22 • Number of events 2 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Cardiac disorders
Pericarditis
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Hospitalization due to persistent fever
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Respiratory, thoracic and mediastinal disorders
Hospitalization due to pneumonia with hypoxia
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Vascular disorders
PRES with intercerebral hemorrhage associated with severe hypertension
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Surgical and medical procedures
Prograf toxicity
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Blood and lymphatic system disorders
Hospitalization due to progressive respiratory distress with hypoxemia
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Blood and lymphatic system disorders
Removal of central venous catheter due to possible thrombus
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Blood and lymphatic system disorders
Respiratory distress secondary to large pharyngeal blood clot
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Infections and infestations
Hospitalization due to seizure and infection
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Gastrointestinal disorders
Hospitalization due to severe nausea, vomiting and diarrhea
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Renal and urinary disorders
Urinary retention, dysuria, and hesitancy
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Vascular disorders
Vascular disorder with facial swelling
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
Other adverse events
| Measure |
Bone Marrow Transplant Recipients
n=22 participants at risk
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
|
|---|---|
|
Immune system disorders
Alopecia
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Anorexia
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Psychiatric disorders
Anxiety
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Respiratory, thoracic and mediastinal disorders
Bronchiolitis obliterans
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Infections and infestations
Catheter related infection
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Nervous system disorders
Cranial Bell's Palsy
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Deep bone pain
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Endocrine disorders
Diabetes mellitus
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Gastrointestinal disorders
Diarrhea
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Hepatobiliary disorders
Elevated alkaline phosphatase
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Hepatobiliary disorders
Elevated alanine transaminase
|
9.1%
2/22 • Number of events 2 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Extremity pain
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Fever
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Headache
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Blood and lymphatic system disorders
Reduced hemoglobin
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Blood and lymphatic system disorders
Central nervous system hemorrhage
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Endocrine disorders
Hyperglycemia
|
9.1%
2/22 • Number of events 2 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Hyperkalemia
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Vascular disorders
Hypertension
|
13.6%
3/22 • Number of events 4 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Hypocalcemia
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Hypokalemia
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Hypomagnesemia
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Respiratory, thoracic and mediastinal disorders
Idiopathic pneumonia syndrome
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Hepatobiliary disorders
Increased liver function tests
|
4.5%
1/22 • Number of events 2 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Infections and infestations
Infection
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Lethargy and confusion
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Nervous system disorders
Neuropathic extremity pain
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Surgical and medical procedures
Post transplant lymphoproliferative disorder
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Surgical and medical procedures
Prograf toxicity
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Vascular disorders
Pulmonary hypertension
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Rectal pain
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Renal and urinary disorders
Renal failure
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Renal and urinary disorders
Renal insufficiency
|
9.1%
2/22 • Number of events 4 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Severe itching
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Severe pain
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Somnolence, depressed level of consciousness
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Gastrointestinal disorders
Stomatitis
|
31.8%
7/22 • Number of events 10 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
General disorders
Tremors
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Renal and urinary disorders
Urinary disorder
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Reproductive system and breast disorders
Vaginal pain
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
|
Infections and infestations
Increased white blood cell count
|
4.5%
1/22 • Number of events 1 • Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place