Pretransplant myeloid and immune suppression, reduced toxicity conditioning with posttransplant cyclophosphamide: Initial outcomes of novel approach for matched unrelated donor hematopoietic stem cell transplant for hemoglobinopathies. Issue 4 (20th January 2021)

• Record Type: Journal Article
• Authors: Kharya, Gaurav; Bakane, Atish N.; Rauthan, Archana M.
• Is Part Of: Pediatric blood & cancer. Volume 68:Issue 4(2021)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2021-01-20
• Subjects: hematopoietic stem cell transplant -- hemoglobinopathies -- matched unrelated donor -- posttransplant cyclophosphamide -- pretransplant immune suppression; Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Cancer in children -- Periodicals; 618.92
• DOI: 10.1002/pbc.28909
• Languages: English
• ISSNs: 1545-5009

Abstract

Abstract: Hematopoietic stem cell transplant (HSCT) is currently the only curative option for thalassemia major (TM) and sickle cell disease (SCD). We report our experience of using pretransplant immune suppression (PTIS), augmented Johns Hopkins conditioning, and posttransplant cyclophosphamide (PTCy) as graft‐versus‐host disease (GvHD) prophylaxis for matched unrelated donor (MUD) transplant in TM/SCD. At a median follow‐up of 307.5 days (range 251‐395), all patients (three TM, one SCD) are alive and disease free. MUD HSCT with PTIS, augmented Johns Hopkins conditioning, and PTCy as GvHD prophylaxis is a promising way of treating patients with hemoglobinopathies with low regimen‐related toxicity (RRT), no risk of graft failure (GF) and minimal GvHD rates.