Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia

Phase 2 Completed 18 enrolled Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

NaSingle-groupOpen-labelTreatment

Summary

Our primary objective is to determine if it is feasible for SAA patients to be transplanted using non-myeloablative conditioning and post transplantation cyclophosphamide with partially HLA-mismatched donors.

Timeline

Start: 2014-08
Primary completion: 2021-12
Completion: 2021-12

Publications

DeZern AE, Zahurak ML, Symons HJ, Cooke KR, Rosner GL, Gladstone DE, Huff CA, Swinnen LJ, Imus P, Borrello I, Wagner-Johnston N, Ambinder RF, Luznik L, Bolanos-Meade J, Fuchs EJ, Jones RJ, Brodsky RA. Haploidentical BMT for severe aplastic anemia with intensive GVHD prophylaxis including posttransplant cyclophosphamide. Blood Adv. 2020 Apr 28;4(8):1770-1779. doi: 10.1182/bloodadvances.2020001729.

Drugs

Evaluation	Drug	Modality	Dose	Route
Background	2-MERCAPTOETHANESULFONIC ACID	Small molecule	40 mg/kg	Intravenous
Background	Cyclophosphamide	Other / unclassified	14.5 mg/kg	Intravenous
Background	Fludarabine	Small molecule	30 mg/m2	Intravenous
Background	Mycophenolic acid mofetil	Unknown	15 mg/kg	Oral
Background	Tacrolimus	Small molecule	—	Oral
Background	Thymoglobulin	Protein / enzyme biologic	2 mg/kg	Intravenous

Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia

Summary

Timeline

Publications

Drugs

Indications