Trial Outcomes & Findings for Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia (NCT NCT00471848)

Last Updated: 2023-09-26

Results Overview

Complete Response (CR) defined as: Haemoglobin normal for age and gender, neutrophils \> 1.5 x 10E9/l, platelets \> 150 x 10E9/l. Partial Response (PR) defined as: * transfusion independence (if previously dependent) or * doubling or normalisation of at least one cell line or * increase of baseline haemoglobin of \> 3 g/dl (if initially \<6) + neutrophils of \> 0.5 x 10E9/l + platelets of \> 20 x 10E9/l (if initially \< 20) No Response (MR) is defined as: worse or not meeting criteria above

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

35 participants

Primary outcome timeframe

at 6months

Results posted on

2023-09-26

Participant Flow

14 sites in 6 countries were open to include patients; in the end 10 sites entered all 35 patients. First Patient In (FPI) 04-Aug-2008, Last Patient In (LPI) 30-Sep-2010

Naive aplastic anaemia patients

Participant milestones

Participant milestones
Measure	Treatment Arm Antithymocyte globulin with cyclosporin in first line treatment of patients with acquired severe aplastic anaemia and patients with non-severe aplastic anaemia who are transfusion dependent; the results were compared with historical controls from the European Group for Blood and Marrow Transplantation (EBMT) database. Patients were matched for age and disease status (NSAA, SAA, VSAA)
Overall Study STARTED	35
Overall Study COMPLETED	35
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment Arm n=35 Participants Antithymocyte globulin with cyclosporin in first line treatment of patients with acquired severe aplastic anaemia and patients with non-severe aplastic anaemia who are transfusion dependent; the results were compared with historical controls from the EBMT database. Patients were matched for age and disease status (NSAA, SAA, VSAA)
Age, Categorical <=18 years	2 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	30 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants

PRIMARY outcome

Timeframe: at 6months

Outcome measures

Outcome measures
Measure	Treatment Arm n=35 Participants Antithymocyte globulin with cyclosporin in first line treatment of patients with acquired severe aplastic anaemia and patients with non-severe aplastic anaemia who are transfusion dependent rabbit antithymocyte globulin: 1.5 vials/10kg daily for 5 days
Number of Participants With Response to Rabbit Antithymocyte Globulin (Thymoglobuline) CR	1 Participants
Number of Participants With Response to Rabbit Antithymocyte Globulin (Thymoglobuline) PR	11 Participants
Number of Participants With Response to Rabbit Antithymocyte Globulin (Thymoglobuline) NR	18 Participants
Number of Participants With Response to Rabbit Antithymocyte Globulin (Thymoglobuline) Transplanted	3 Participants
Number of Participants With Response to Rabbit Antithymocyte Globulin (Thymoglobuline) Dead	2 Participants

SECONDARY outcome

Timeframe: at 2 years

Failure free survival is defined as a failure of the protocol: no achievement of response, relapse, disease progression requiring a second course of immune suppressive therapy (IST) or a stem cell transplant, death, or later clonal disorders such as Paroxysmal Nocturnal Hemoglobinuria (PNH), Myelodysplastic Syndrome (MDS) and Acute Myeloblastic Leukemia (AML).

Outcome measures

Outcome data not reported

Adverse Events

Experimental: Treament Arm

Serious events: 27 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Experimental: Treament Arm n=35 participants at risk Antithymocyte globulin with cyclosporin in first line treatment of patients with acquired severe aplastic anaemia and patients with non-severe aplastic anaemia who are transfusion dependent
Gastrointestinal disorders Pyrexia	2.9% 1/35 • Number of events 1
Gastrointestinal disorders Abdominal Pain	2.9% 1/35 • Number of events 1
Immune system disorders Allergic Reaction / Hypersensitivy	2.9% 1/35 • Number of events 1
Infections and infestations Bacterial Infections	2.9% 1/35 • Number of events 1
Respiratory, thoracic and mediastinal disorders Bilateral Basal Pneumonia	2.9% 1/35 • Number of events 1
Infections and infestations Blood-reactivation	2.9% 1/35 • Number of events 1
Cardiac disorders Cardiac Arrest	2.9% 1/35 • Number of events 1
Infections and infestations Coccy geal fistula abscessed	2.9% 1/35 • Number of events 1
Infections and infestations Colonisation in Staphylococcus aures methicillin resistant	2.9% 1/35 • Number of events 1
General disorders Death	17.1% 6/35 • Number of events 6
General disorders Disease progression	5.7% 2/35 • Number of events 2
Gastrointestinal disorders Duodenal Ulcer	2.9% 1/35 • Number of events 1
Respiratory, thoracic and mediastinal disorders Dyspnea	2.9% 1/35 • Number of events 1
Infections and infestations Facial Infection	2.9% 1/35 • Number of events 1
Investigations Elevated ALT levels	2.9% 1/35 • Number of events 1
Gastrointestinal disorders Gastritis	2.9% 1/35 • Number of events 1
Infections and infestations Febrile Neutropenia	14.3% 5/35 • Number of events 6
Investigations Hyper Bilirubinimia	2.9% 1/35 • Number of events 1
Nervous system disorders Headache	2.9% 1/35 • Number of events 1
Infections and infestations Klebsiella Septicaemia	2.9% 1/35 • Number of events 1
Infections and infestations Hickmann Line Infection	2.9% 1/35 • Number of events 1
Infections and infestations Infection	5.7% 2/35 • Number of events 2
Infections and infestations Neutropenic sepsis	14.3% 5/35 • Number of events 5
Infections and infestations Neutropenic Fever	2.9% 1/35 • Number of events 1
Infections and infestations Norovirus infection	2.9% 1/35 • Number of events 1
General disorders Pyrexia with rigours	2.9% 1/35 • Number of events 1
Endocrine disorders Pancreatic endocrine, glucose intolerance	2.9% 1/35 • Number of events 1
Metabolism and nutrition disorders Renal Impairment	2.9% 1/35 • Number of events 1
Eye disorders Retinopathy	2.9% 1/35 • Number of events 1
Nervous system disorders Seizures	2.9% 1/35 • Number of events 1
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Secondary Clonal Malignancy	2.9% 1/35 • Number of events 1
Infections and infestations Sepsis	8.6% 3/35 • Number of events 4
Immune system disorders Serum Sickness	5.7% 2/35 • Number of events 2
General disorders Unrelated Donor Stem Cell Transplant	2.9% 1/35 • Number of events 1
Cardiac disorders Vasovagal Episode	2.9% 1/35 • Number of events 1
Metabolism and nutrition disorders Cholestasis	2.9% 1/35 • Number of events 1

Other adverse events

Adverse event data not reported

Additional Information

Prof. Judith Marsh

European Group for Blood and Marrow Transplantation

Phone: +31 (0)71 5265005

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Participating PIs can not publish on the patients they have included in this prospective trial before the results of the trial have been published in a peer-reviewed journal.
Publication restrictions are in place

Restriction type: OTHER