Unmanipulated haploidentical transplantation conditioning with busulfan, cyclophosphamide and anti-thymoglobulin for adult severe aplastic anaemia

• Published in: Bone marrow transplantation (Basingstoke) 2018-02, Vol.53 (2), p.188-192
• Main Authors: Xu, L-P, Xu, Z-L, Wang, F-R, Mo, X-D, Han, T-T, Han, W, Chen, Y-H, Zhang, Y-Y, Wang, J-Z, Wang, Y, Yan, C-H, Sun, Y-Q, Tang, F-F, Zhang, X-H, Huang, X-J
• Format: Article
• Language: English
• Subjects: 692/699/1541/13; Adult; Adults; Anemia; Anemia, Aplastic - drug therapy; Anemia, Aplastic - pathology; Antilymphocyte Serum - pharmacology; Antilymphocyte Serum - therapeutic use; Aplastic anemia; Bone marrow; Bone marrow transplantation; Busulfan; Busulfan - pharmacology; Busulfan - therapeutic use; Care and treatment; Cell Biology; Conditioning; Cyclophosphamide; Cyclophosphamide - pharmacology; Cyclophosphamide - therapeutic use; Diagnosis; Dosage and administration; Engraftment; Female; Graft-versus-host reaction; Hematology; Hematopoietic stem cell transplantation; Hematopoietic Stem Cell Transplantation - methods; Humans; Immunosuppressive agents; Incidence; Internal Medicine; Male; Medicine; Medicine & Public Health; Multivariate analysis; original-article; Patients; Peripheral blood; Public Health; Quality; Retrospective Studies; Stem cell transplantation; Stem Cells; Survival; Thymoglobulin; Transplantation; Transplantation Conditioning - methods; Transplantation, Haploidentical - methods
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/bmt.2017.237

We conducted a retrospective analysis to evaluate outcomes of haploidentical transplantation in adult severe aplastic anaemia (SAA) patients. Fifty-one adults received haploidentical transplantation between May 2011 and December 2016. Patients were administered busulfan (Bu), cyclophosphamide (Cy) and anti-thymoglobulin (ATG) as conditioning regimens, followed by bone marrow and peripheral blood transplantation. The patients’ median age was 25 years. Forty-nine patients survived for more than 28 days and all achieved donor myeloid engraftment. The median time for myeloid engraftment and platelet recovery was 13 days (range, 10–21) and 17.5 (range, 7–101) days. The cumulative incidence (CI) of grade II–IV and III–IV acute GvHD) was 20.00±0.33% and 6.00±0.12%, respectively. The incidence of chronic GvHD was 14.00±0.36% and 25.90±0.71%, and that of moderate-severe chronic GvHD was 2.51±0.06% and 6.92±0.25% at 1 and 3 years, respectively. The 3-year estimated overall survival and failure-free survival were both 83.5±5.4% with a median follow-up of 21.1 months. Multivariate analysis showed hematopoietic cell transplantation-specific comorbidity index (HCT-CI) score of ⩾3 was significantly associated with worse outcome. Haploidentical transplantation conditioning including Bu/Cy/ATG was a safe and effective strategy for adult SAA patients, and HCT-CI might be an outcome predictor in these patients.