PMID- 29512932 OWN - NLM STAT- MEDLINE DCOM- 20190506 LR - 20190506 IS - 1545-5017 (Electronic) IS - 1545-5009 (Linking) VI - 65 IP - 7 DP - 2018 Jul TI - Modified conditioning regimen improves outcomes of unrelated donor peripheral blood stem cell transplantation for beta-thalassaemia major patients. PG - e27026 LID - 10.1002/pbc.27026 [doi] AB - BACKGROUND: The objective of this study was to evaluate the feasibility of a modified conditioning regimen for the treatment of patients with beta-thalassaemia major (TM), using unrelated donor peripheral blood stem cell transplantation (UD-PBSCT). METHODS: A modified conditioning regimen based on intravenous busulfan, cyclophosphamide, fludarabine, and antithymocyte globulin was performed in 50 consecutive childhood patients with beta-TM and a median age of 4.6 years (range, 2-12 years). According to Pesaro's classification, three classes of risk are identified using the criteria of degree of hepatomegaly, portal fibrosis, and quality of the chelation treatment. Patients with three adverse criteria constituted class III, none of the adverse criteria constituted class I, and one or two of the adverse criteria formed class II. Ten patients were class I, 36 class II, and four class III. All patients were transplanted with UDs containing 37 of 10/10 human leukocyte antigen (HLA)-matched pairs, 11 of 9/10 matched pairs, and two of 8/10 matched pairs. The median follow-up was 36 months (range, 9-96 months). RESULTS: All patients successfully achieved engraftment, two of whom developed persistent thrombocytopaenia. The incidence of acute graft-versus-host disease (aGVHD) grade III-IV and chronic graft-versus-host disease (cGVHD) were 12% and 8%, respectively. However, 8.3% of HLA-matched and 15.4% of HLA-mismatched patients developed aGVHD. The incidence of severe bacterial infections and fungal pneumonia was 12% and 20%, respectively. The 3-year overall survival, disease-free survival, graft rejection, and transplant-related mortality were 94%, 92%, 2%, and 6%, respectively. CONCLUSION: This modified conditioning protocol effectively improved outcomes of UD-PBSCT for patients with beta-TM. CI - (c) 2018 Wiley Periodicals, Inc. FAU - Huang, Ke AU - Huang K AUID- ORCID: 0000-0003-1854-7840 AD - Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, People's Republic of China. AD - Key Laboratory of Malignant Tumor Gene Regulation and Target Therapy of Guangdong Higher Education Institutes, Sun Yat-sen University, Guangzhou, People's Republic of China. FAU - Zhou, Dun-Hua AU - Zhou DH AD - Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, People's Republic of China. AD - Key Laboratory of Malignant Tumor Gene Regulation and Target Therapy of Guangdong Higher Education Institutes, Sun Yat-sen University, Guangzhou, People's Republic of China. FAU - Li, Yang AU - Li Y AD - Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, People's Republic of China. AD - Key Laboratory of Malignant Tumor Gene Regulation and Target Therapy of Guangdong Higher Education Institutes, Sun Yat-sen University, Guangzhou, People's Republic of China. FAU - Xu, Hong-Gui AU - Xu HG AD - Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, People's Republic of China. AD - Key Laboratory of Malignant Tumor Gene Regulation and Target Therapy of Guangdong Higher Education Institutes, Sun Yat-sen University, Guangzhou, People's Republic of China. FAU - Que, Li-Ping AU - Que LP AD - Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, People's Republic of China. AD - Key Laboratory of Malignant Tumor Gene Regulation and Target Therapy of Guangdong Higher Education Institutes, Sun Yat-sen University, Guangzhou, People's Republic of China. FAU - Chen, Chun AU - Chen C AD - Department of Pediatrics, The Seventh Affiliated Hospital of Sun Yat-sen University, Shenzhen, People's Republic of China. FAU - Xue, Hong-Man AU - Xue HM AD - Department of Pediatrics, The Seventh Affiliated Hospital of Sun Yat-sen University, Shenzhen, People's Republic of China. FAU - Guo, Hai-Xia AU - Guo HX AD - Department of Pediatrics, Southern Medical University Nan fang Hospital, Guangzhou, People's Republic of China. FAU - Weng, Wen-Jun AU - Weng WJ AD - Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, People's Republic of China. AD - Key Laboratory of Malignant Tumor Gene Regulation and Target Therapy of Guangdong Higher Education Institutes, Sun Yat-sen University, Guangzhou, People's Republic of China. FAU - Huang, Shao-Liang AU - Huang SL AD - Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, People's Republic of China. AD - Key Laboratory of Malignant Tumor Gene Regulation and Target Therapy of Guangdong Higher Education Institutes, Sun Yat-sen University, Guangzhou, People's Republic of China. FAU - Fang, Jian-Pei AU - Fang JP AD - Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, People's Republic of China. AD - Key Laboratory of Malignant Tumor Gene Regulation and Target Therapy of Guangdong Higher Education Institutes, Sun Yat-sen University, Guangzhou, People's Republic of China. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20180307 PL - United States TA - Pediatr Blood Cancer JT - Pediatric blood & cancer JID - 101186624 RN - 0 (Immunosuppressive Agents) SB - IM MH - Adolescent MH - Child MH - Child, Preschool MH - Female MH - Follow-Up Studies MH - Graft Rejection/drug therapy/etiology/*mortality MH - Graft Survival MH - Graft vs Host Disease/drug therapy/etiology/*mortality MH - Humans MH - Immunosuppressive Agents/therapeutic use MH - Male MH - Peripheral Blood Stem Cell Transplantation/*adverse effects MH - *Transplantation Conditioning MH - Treatment Outcome MH - Unrelated Donors MH - beta-Thalassemia/complications/*therapy OTO - NOTNLM OT - haematopoietic stem cells OT - peripheral blood OT - thalassaemia OT - transplantation OT - unrelated donor EDAT- 2018/03/08 06:00 MHDA- 2019/05/07 06:00 CRDT- 2018/03/08 06:00 PHST- 2017/10/05 00:00 [received] PHST- 2018/01/09 00:00 [revised] PHST- 2018/01/28 00:00 [accepted] PHST- 2018/03/08 06:00 [pubmed] PHST- 2019/05/07 06:00 [medline] PHST- 2018/03/08 06:00 [entrez] AID - 10.1002/pbc.27026 [doi] PST - ppublish SO - Pediatr Blood Cancer. 2018 Jul;65(7):e27026. doi: 10.1002/pbc.27026. Epub 2018 Mar 7.