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Haploidentical BMT for severe aplastic anemia with intensive GVHD prophylaxis including posttransplant cyclophosphamide.
Blood Adv. 2020 Apr 28; 4(8):1770-1779.BA

Abstract

Severe aplastic anemia (SAA) is a stem cell disorder often treated with bone marrow transplantation (BMT) to reconstitute hematopoiesis. Outcomes of related HLA-haploidentical (haplo) donors after reduced-intensity conditioning with intensive graft-versus-host disease (GVHD) prophylaxis including posttransplantation cyclophosphamide are presented here from 37 SAA, 20 relapsed/refractory (R/R), and 17 treatment-naïve (TN) SAA patients. Median follow-up is 32 months (90% confidence interval [CI], 29-44). The median age was 25 (range, 4-69) years. The median time to neutrophil recovery was 17 days (range, 15-88). Four of 37 patients (11%) experienced graft failure (GF). There was 1 primary GF of 20 patients in the R/R group and 3 of 17 in the TN group at 200 cGy (1 primary, 2 secondary), but none in the 10 patients who received 400 cGy total body irradiation. Two patients with GF succumbed to infection and 2 were rescued with second haplo BMT. The overall survival for all patients is 94% (90% CI, 88-100) at 1 and 2 years. The cumulative incidence of grade II-IV acute GVHD at day 100 is 11%. The cumulative index of chronic GVHD at 2 years is 8%. Similar results were seen in 10 SAA patients who received the identical nonmyeloablative regimen with posttransplant cyclophosphamide but matched donor transplants. Haplo BMT with posttransplant cyclophosphamide represents a potential cure in SAA, with all 20 R/R currently alive, disease-free, and with no evidence of active GVHD. Extending this approach to TN patients was associated with higher GF rates, but an increase in total body irradiation dose to 400 cGy was associated with durable engraftment without greater early toxicity. Nonmyeloablative haplo BMT in TN SAA could lead to a paradigm shift, such that essentially all patients can proceed quickly to safe, curative BMT. These trials were registered at www.cincialtrials.gov as #NCT02224872) and #NCT02833805.

Authors+Show Affiliations

Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD. Division of Hematology, Department of Medicine, Johns Hopkins University, Baltimore, MD; and.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD. Department of Oncology Biostatistics and.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD. Division of Pediatric Oncology, Sidney Kimmel Cancer Center, Baltimore, MD.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD. Division of Pediatric Oncology, Sidney Kimmel Cancer Center, Baltimore, MD.Department of Oncology Biostatistics and.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD. Division of Hematology, Department of Medicine, Johns Hopkins University, Baltimore, MD; and.Department of Oncology, Sidney Kimmel Cancer Center, Baltimore, MD. Division of Hematology, Department of Medicine, Johns Hopkins University, Baltimore, MD; and.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

32343796

Citation

DeZern, Amy E., et al. "Haploidentical BMT for Severe Aplastic Anemia With Intensive GVHD Prophylaxis Including Posttransplant Cyclophosphamide." Blood Advances, vol. 4, no. 8, 2020, pp. 1770-1779.
DeZern AE, Zahurak ML, Symons HJ, et al. Haploidentical BMT for severe aplastic anemia with intensive GVHD prophylaxis including posttransplant cyclophosphamide. Blood Adv. 2020;4(8):1770-1779.
DeZern, A. E., Zahurak, M. L., Symons, H. J., Cooke, K. R., Rosner, G. L., Gladstone, D. E., Huff, C. A., Swinnen, L. J., Imus, P., Borrello, I., Wagner-Johnston, N., Ambinder, R. F., Luznik, L., Bolaños-Meade, J., Fuchs, E. J., Jones, R. J., & Brodsky, R. A. (2020). Haploidentical BMT for severe aplastic anemia with intensive GVHD prophylaxis including posttransplant cyclophosphamide. Blood Advances, 4(8), 1770-1779. https://doi.org/10.1182/bloodadvances.2020001729
DeZern AE, et al. Haploidentical BMT for Severe Aplastic Anemia With Intensive GVHD Prophylaxis Including Posttransplant Cyclophosphamide. Blood Adv. 2020 Apr 28;4(8):1770-1779. PubMed PMID: 32343796.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Haploidentical BMT for severe aplastic anemia with intensive GVHD prophylaxis including posttransplant cyclophosphamide. AU - DeZern,Amy E, AU - Zahurak,Marianna L, AU - Symons,Heather J, AU - Cooke,Kenneth R, AU - Rosner,Gary L, AU - Gladstone,Douglas E, AU - Huff,Carol Ann, AU - Swinnen,Lode J, AU - Imus,Philip, AU - Borrello,Ivan, AU - Wagner-Johnston,Nina, AU - Ambinder,Richard F, AU - Luznik,Leo, AU - Bolaños-Meade,Javier, AU - Fuchs,Ephraim J, AU - Jones,Richard J, AU - Brodsky,Robert A, PY - 2020/02/24/received PY - 2020/03/25/accepted PY - 2020/4/29/entrez PY - 2020/4/29/pubmed PY - 2020/4/29/medline SP - 1770 EP - 1779 JF - Blood advances JO - Blood Adv VL - 4 IS - 8 N2 - Severe aplastic anemia (SAA) is a stem cell disorder often treated with bone marrow transplantation (BMT) to reconstitute hematopoiesis. Outcomes of related HLA-haploidentical (haplo) donors after reduced-intensity conditioning with intensive graft-versus-host disease (GVHD) prophylaxis including posttransplantation cyclophosphamide are presented here from 37 SAA, 20 relapsed/refractory (R/R), and 17 treatment-naïve (TN) SAA patients. Median follow-up is 32 months (90% confidence interval [CI], 29-44). The median age was 25 (range, 4-69) years. The median time to neutrophil recovery was 17 days (range, 15-88). Four of 37 patients (11%) experienced graft failure (GF). There was 1 primary GF of 20 patients in the R/R group and 3 of 17 in the TN group at 200 cGy (1 primary, 2 secondary), but none in the 10 patients who received 400 cGy total body irradiation. Two patients with GF succumbed to infection and 2 were rescued with second haplo BMT. The overall survival for all patients is 94% (90% CI, 88-100) at 1 and 2 years. The cumulative incidence of grade II-IV acute GVHD at day 100 is 11%. The cumulative index of chronic GVHD at 2 years is 8%. Similar results were seen in 10 SAA patients who received the identical nonmyeloablative regimen with posttransplant cyclophosphamide but matched donor transplants. Haplo BMT with posttransplant cyclophosphamide represents a potential cure in SAA, with all 20 R/R currently alive, disease-free, and with no evidence of active GVHD. Extending this approach to TN patients was associated with higher GF rates, but an increase in total body irradiation dose to 400 cGy was associated with durable engraftment without greater early toxicity. Nonmyeloablative haplo BMT in TN SAA could lead to a paradigm shift, such that essentially all patients can proceed quickly to safe, curative BMT. These trials were registered at www.cincialtrials.gov as #NCT02224872) and #NCT02833805. SN - 2473-9537 UR - https://www.unboundmedicine.com/medline/citation/32343796/Haploidentical_BMT_for_severe_aplastic_anemia_with_intensive_GVHD_prophylaxis_including_posttransplant_cyclophosphamide L2 - https://ashpublications.org/bloodadvances/article-lookup/doi/10.1182/bloodadvances.2020001729 DB - PRIME DP - Unbound Medicine ER -
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