drugset / Trial / NCT03608059
ATG/PTCy in Haplo-PBSCT Randomized Controlled,Multi-center
Phase 4
Completed
418 enrolled
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
RandomizedSingle-groupSingle-blindTreatment
Summary
A novel regimen, which is composed of a low dose of ATG (5 mg/kg) and low-dose PTCy (one dose of PTCy, 50mg/kg) for GvHD prophylaxis in Haplo-PBSCT for patients with hematologic malignancies, was designed to decrease the risk of aGvHD and lower the incidence of virus reactivation.
Timeline
- Start
- 2018-07-28
- Primary completion
- 2023-08-23
- Completion
- 2023-08-23
Outcome
Missed primary endpoint
paper By day +100, the CI of grade 2 to 4 aGVHD did not differ significantly among the 3 groups (P = .210). PMID 41849227 ↗
paper No significant differences were observed in survival outcomes among the 3 groups. PMID 41849227 ↗
Publications
- Background Luznik L, Jalla S, Engstrom LW, Iannone R, Fuchs EJ. Durable engraftment of major histocompatibility complex-incompatible cells after nonmyeloablative conditioning with fludarabine, low-dose total body irradiation, and posttransplantation cyclophosphamide. Blood. 2001 Dec 1;98(12):3456-64. doi: 10.1182/blood.v98.12.3456.
- Background Wang Y, Liu DH, Liu KY, Xu LP, Zhang XH, Han W, Chen H, Chen YH, Wang FR, Wang JZ, Sun YQ, Huang XJ. Long-term follow-up of haploidentical hematopoietic stem cell transplantation without in vitro T cell depletion for the treatment of leukemia: nine years of experience at a single center. Cancer. 2013 Mar 1;119(5):978-85. doi: 10.1002/cncr.27761. Epub 2012 Oct 23.
- Background Wang Y, Chang YJ, Xu LP, Liu KY, Liu DH, Zhang XH, Chen H, Han W, Chen YH, Wang FR, Wang JZ, Chen Y, Yan CH, Huo MR, Li D, Huang XJ. Who is the best donor for a related HLA haplotype-mismatched transplant? Blood. 2014 Aug 7;124(6):843-50. doi: 10.1182/blood-2014-03-563130. Epub 2014 Jun 10.
- Background Liu Q, Xuan L, Liu H, Huang F, Zhou H, Fan Z, Zhao K, Wu M, Xu L, Zhai X, Zhang F, Liu C, Sun J, Huang X. Molecular monitoring and stepwise preemptive therapy for Epstein-Barr virus viremia after allogeneic stem cell transplantation. Am J Hematol. 2013 Jul;88(7):550-5. doi: 10.1002/ajh.23452. Epub 2013 May 30.
- Background Tischer J, Engel N, Fritsch S, Prevalsek D, Hubmann M, Schulz C, Zoellner AK, Bucklein V, Reibke R, Mumm F, Rieger CT, Hill W, Ledderose G, Stemmler HJ, Kohnke T, Jager G, Kolb HJ, Schmid C, Moosmann A, Hausmann A. Virus infection in HLA-haploidentical hematopoietic stem cell transplantation: incidence in the context of immune recovery in two different transplantation settings. Ann Hematol. 2015 Oct;94(10):1677-88. doi: 10.1007/s00277-015-2423-y. Epub 2015 Jun 10.
- Background Luznik L, O'Donnell PV, Symons HJ, Chen AR, Leffell MS, Zahurak M, Gooley TA, Piantadosi S, Kaup M, Ambinder RF, Huff CA, Matsui W, Bolanos-Meade J, Borrello I, Powell JD, Harrington E, Warnock S, Flowers M, Brodsky RA, Sandmaier BM, Storb RF, Jones RJ, Fuchs EJ. HLA-haploidentical bone marrow transplantation for hematologic malignancies using nonmyeloablative conditioning and high-dose, posttransplantation cyclophosphamide. Biol Blood Marrow Transplant. 2008 Jun;14(6):641-50. doi: 10.1016/j.bbmt.2008.03.005.
- Background Ruggeri A, Labopin M, Bacigalupo A, Gulbas Z, Koc Y, Blaise D, Bruno B, Irrera G, Tischer J, Diez-Martin JL, Castagna L, Ciceri F, Mohty M, Nagler A. Bone marrow versus mobilized peripheral blood stem cells in haploidentical transplants using posttransplantation cyclophosphamide. Cancer. 2018 Apr 1;124(7):1428-1437. doi: 10.1002/cncr.31228. Epub 2018 Jan 23.
- Background Bashey A, Zhang MJ, McCurdy SR, St Martin A, Argall T, Anasetti C, Ciurea SO, Fasan O, Gaballa S, Hamadani M, Munshi P, Al Malki MM, Nakamura R, O'Donnell PV, Perales MA, Raj K, Romee R, Rowley S, Rocha V, Salit RB, Solh M, Soiffer RJ, Fuchs EJ, Eapen M. Mobilized Peripheral Blood Stem Cells Versus Unstimulated Bone Marrow As a Graft Source for T-Cell-Replete Haploidentical Donor Transplantation Using Post-Transplant Cyclophosphamide. J Clin Oncol. 2017 Sep 10;35(26):3002-3009. doi: 10.1200/JCO.2017.72.8428. Epub 2017 Jun 23.
- Background O'Donnell PV, Luznik L, Jones RJ, Vogelsang GB, Leffell MS, Phelps M, Rhubart P, Cowan K, Piantados S, Fuchs EJ. Nonmyeloablative bone marrow transplantation from partially HLA-mismatched related donors using posttransplantation cyclophosphamide. Biol Blood Marrow Transplant. 2002;8(7):377-86. doi: 10.1053/bbmt.2002.v8.pm12171484.
- Background Brunstein CG, Gutman JA, Weisdorf DJ, Woolfrey AE, Defor TE, Gooley TA, Verneris MR, Appelbaum FR, Wagner JE, Delaney C. Allogeneic hematopoietic cell transplantation for hematologic malignancy: relative risks and benefits of double umbilical cord blood. Blood. 2010 Nov 25;116(22):4693-9. doi: 10.1182/blood-2010-05-285304. Epub 2010 Aug 4.
- Background Chen J, Wang RX, Chen F, Sun AN, Qiu HY, Jin ZM, Tang XW, Han Y, Fu ZZ, He GS, Miao M, Ma X, Wu DP. Combination of a haploidentical SCT with an unrelated cord blood unit: a single-arm prospective study. Bone Marrow Transplant. 2014 Feb;49(2):206-11. doi: 10.1038/bmt.2013.154. Epub 2013 Oct 21.
- Background Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J, Thomas ED. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant. 1995 Jun;15(6):825-8.
- Background Martin PJ, Lee SJ, Przepiorka D, Horowitz MM, Koreth J, Vogelsang GB, Walker I, Carpenter PA, Griffith LM, Akpek G, Mohty M, Wolff D, Pavletic SZ, Cutler CS. National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: VI. The 2014 Clinical Trial Design Working Group Report. Biol Blood Marrow Transplant. 2015 Aug;21(8):1343-59. doi: 10.1016/j.bbmt.2015.05.004. Epub 2015 May 15.
- Yang J, Jia Y, Hu X, Zhou F, Ni X, Wan J, Ding Y, Kang M, Yu X, Jiang C, Wang L, Wan L, Cai Y, Huang C, Qiu H, Ding X, Tong Y, Dong B, Zhou K, Song X. A randomized trial of GVHD prophylaxis in haploidentical PBSC transplantation: ATG, PTCy, and low-dose combination therapy. Blood. 2026 Jun 25;147(26):3168-3178. doi: 10.1182/blood.2025032569.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE) | Protein / enzyme biologic | 2.5 mg/kg | — |
| Subject | Cyclophosphamide | Other / unclassified | 50 mg/kg | — |