1. Allogenic peripheral stem cell transplantation from HLA-matched related donors for adult sickle cell disease: remarkable outcomes from a single-center trial
- Author
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Çiğdem Gereklioğlu, Cagla Sariturk, Mahmut Yeral, Can Boga, Pelin Aytan, Nurhilal Büyükkurt, Soner Solmaz, Aslı Korur, Hakan Ozdogu, Ilknur Kozanoglu, Süheyl Asma, and Mutlu Kasar
- Subjects
Adult ,Male ,medicine.medical_specialty ,Anemia, Sickle Cell ,Single Center ,Gastroenterology ,Young Adult ,03 medical and health sciences ,0302 clinical medicine ,Internal medicine ,Humans ,Transplantation, Homologous ,Medicine ,Retrospective Studies ,Peripheral Blood Stem Cell Transplantation ,Transplantation ,business.industry ,Hematology ,Middle Aged ,Total body irradiation ,medicine.disease ,Tissue Donors ,Fludarabine ,Peripheral stem cell transplantation ,surgical procedures, operative ,Graft-versus-host disease ,030220 oncology & carcinogenesis ,Female ,Stem cell ,business ,Busulfan ,030215 immunology ,medicine.drug - Abstract
Adult patients with sickle cell disease (SCD) are highly susceptible to stem cell transplant complications, including drug toxicity, graft versus host disease (GVHD), and graft rejection due to SCD-related tissue damage, endothelial activation, and inflammation. The scarcity of compatible stem cells for transplantation further limits treatment options, with only 43 cases of adult allogeneic peripheral blood stem cell transplantation (allo-PSCT) from human leukocyte antigen (HLA)-identical sibling donors reported in the international registry for the period 1986-2013. Herein we report remarkable outcomes in a cohort of adult SCD patients who underwent allo-PSCT using a fludarabine (Flu), busulfan (Bu), and anti-T-cell lymphocyte globulin (ATG)-based conditioning regimen in combination with very low dose total body irradiation (TBI), followed by post-transplant cyclophosphamide (Cy) and sirolimus as GVHD prophylaxis. We performed a single-center, retrospective study consisting of 20 consecutive patients (mean age 33.4 years) who underwent allo-PSCT from HLA-matched related donors with a conditioning regimen of Flu 150/Bu 3.2/Cy 29/ATG 30 (Fresenius)/TBI 200 between September 2013 and September 2017. Data were validated by an independent data audit group of the affiliated JACIE-accredited transplantation center. All patients experienced a sustained donor cell engraftment. Full donor chimerism (total cell) occurred within 180 days in all patients. Mean duration of follow-up was 13.8 months (range: 0.3-50 months), with 12 (60%) patients completing 12 months. No non-relapse mortality or graft rejection occurred. Successful treatment was achieved without the presence of graft loss, grade III-IV acute GVHD, extensive chronic GVHD, or other major complications. Allo-PSCT in combination with Flu 150/Bu 3.2/Cy 29/ATG 30(Fresenius)/TBI 200- Cy/Sirolimus therapy yielded encouraging outcomes with no mortality and low incidence of GVHD. Further controlled studies will be necessary to compare transplant protocols and long-term outcomes.
- Published
- 2018
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