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Results of a 2-arm, phase 2 clinical trial using post-transplantation cyclophosphamide for the prevention of graft-versus-host disease in haploidentical donor and mismatched unrelated donor hematopoietic stem cell transplantation.
Cancer. 2016 Nov 15; 122(21):3316-3326.C

Abstract

BACKGROUND

High-dose, post-transplantation cyclophosphamide (PTCy) to prevent graft-versus-host disease (GVHD) has improved outcomes in haploidentical (HAPLO) stem cell transplantation (SCT). However, it remains unclear whether this strategy is effective in SCT from 1-antigen human leukocyte antigen (HLA)-mismatched unrelated donors (9/10 MUD) and how the outcomes of these patients compare with those of haploidentical transplantation recipients.

METHODS

A parallel, 2-arm, nonrandomized phase 2 clinical trial was conducted of melphalan-based reduced-intensity conditioning with PTCy, tacrolimus, and mycophenolate mofetil to prevent GVHD in patients with high-risk hematologic malignancies who underwent HAPLO (n = 60) or 9/10 MUD (n = 46) SCT.

RESULTS

The 1-year overall and progression-free survival rates were 70% and 60%, respectively, in the HAPLO arm and 60% and 47%, respectively, in the 9/10 MUD arm. The day +100 cumulative incidence of grade II to IV acute GVHD and grade III to IV acute GVHD was 28% and 3%, respectively, in the HAPLO arm and 33% and 13%, respectively, in the 9/10 MUD arm. The 2-year cumulative incidence of chronic GVHD was 24% in the HAPLO arm and 19% in the 9/10 MUD arm. The 1-year cumulative incidence of nonrelapse mortality was 21% in the HAPLO arm and 31% in the 9/10 MUD arm, and the 1-year relapse rate was 19% in the HAPLO arm and 25% in the 9/10 MUD arm.

CONCLUSIONS

Although this was a nonrandomized study and could not serve as a direct comparison between the 2 groups, the authors conclude that PTCy-based GVHD prophylaxis is effective for both HAPLO and 9/10 MUD SCTs. Prospective randomized trials will be required to compare the efficacies of alternative donor options for patients lacking HLA-matched donors. Cancer 2016;122:3316-3326. © 2016 American Cancer Society.

Authors+Show Affiliations

Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.University Medical Center Freiburg, Freiburg, Germany.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Hematopathology, The University of Texas MD Anderson Cancer Center, Houston, Texas.Division of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, Texas.Division of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Laboratory Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas. sciurea@mdanderson.org.

Pub Type(s)

Clinical Trial, Phase II
Comparative Study
Journal Article

Language

eng

PubMed ID

27404668

Citation

Gaballa, Sameh, et al. "Results of a 2-arm, Phase 2 Clinical Trial Using Post-transplantation Cyclophosphamide for the Prevention of Graft-versus-host Disease in Haploidentical Donor and Mismatched Unrelated Donor Hematopoietic Stem Cell Transplantation." Cancer, vol. 122, no. 21, 2016, pp. 3316-3326.
Gaballa S, Ge I, El Fakih R, et al. Results of a 2-arm, phase 2 clinical trial using post-transplantation cyclophosphamide for the prevention of graft-versus-host disease in haploidentical donor and mismatched unrelated donor hematopoietic stem cell transplantation. Cancer. 2016;122(21):3316-3326.
Gaballa, S., Ge, I., El Fakih, R., Brammer, J. E., Kongtim, P., Tomuleasa, C., Wang, S. A., Lee, D., Petropoulos, D., Cao, K., Rondon, G., Chen, J., Hammerstrom, A., Lombardi, L., Alatrash, G., Korbling, M., Oran, B., Kebriaei, P., Ahmed, S., ... Ciurea, S. O. (2016). Results of a 2-arm, phase 2 clinical trial using post-transplantation cyclophosphamide for the prevention of graft-versus-host disease in haploidentical donor and mismatched unrelated donor hematopoietic stem cell transplantation. Cancer, 122(21), 3316-3326. https://doi.org/10.1002/cncr.30180
Gaballa S, et al. Results of a 2-arm, Phase 2 Clinical Trial Using Post-transplantation Cyclophosphamide for the Prevention of Graft-versus-host Disease in Haploidentical Donor and Mismatched Unrelated Donor Hematopoietic Stem Cell Transplantation. Cancer. 2016 Nov 15;122(21):3316-3326. PubMed PMID: 27404668.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Results of a 2-arm, phase 2 clinical trial using post-transplantation cyclophosphamide for the prevention of graft-versus-host disease in haploidentical donor and mismatched unrelated donor hematopoietic stem cell transplantation. AU - Gaballa,Sameh, AU - Ge,Isabell, AU - El Fakih,Riad, AU - Brammer,Jonathan E, AU - Kongtim,Piyanuch, AU - Tomuleasa,Ciprian, AU - Wang,Sa A, AU - Lee,Dean, AU - Petropoulos,Demetrios, AU - Cao,Kai, AU - Rondon,Gabriela, AU - Chen,Julianne, AU - Hammerstrom,Aimee, AU - Lombardi,Lindsey, AU - Alatrash,Gheath, AU - Korbling,Martin, AU - Oran,Betul, AU - Kebriaei,Partow, AU - Ahmed,Sairah, AU - Shah,Nina, AU - Rezvani,Katayoun, AU - Marin,David, AU - Bashir,Qaiser, AU - Alousi,Amin, AU - Nieto,Yago, AU - Qazilbash,Muzaffar, AU - Hosing,Chitra, AU - Popat,Uday, AU - Shpall,Elizabeth J, AU - Khouri,Issa, AU - Champlin,Richard E, AU - Ciurea,Stefan O, Y1 - 2016/07/12/ PY - 2016/03/12/received PY - 2016/05/17/revised PY - 2016/05/24/accepted PY - 2016/10/21/pubmed PY - 2017/5/26/medline PY - 2016/7/13/entrez KW - 9/10 matched unrelated donors KW - graft-versus-host disease KW - haploidentical transplantation KW - hematologic malignancies KW - post-transplantation cyclophosphamide SP - 3316 EP - 3326 JF - Cancer JO - Cancer VL - 122 IS - 21 N2 - BACKGROUND: High-dose, post-transplantation cyclophosphamide (PTCy) to prevent graft-versus-host disease (GVHD) has improved outcomes in haploidentical (HAPLO) stem cell transplantation (SCT). However, it remains unclear whether this strategy is effective in SCT from 1-antigen human leukocyte antigen (HLA)-mismatched unrelated donors (9/10 MUD) and how the outcomes of these patients compare with those of haploidentical transplantation recipients. METHODS: A parallel, 2-arm, nonrandomized phase 2 clinical trial was conducted of melphalan-based reduced-intensity conditioning with PTCy, tacrolimus, and mycophenolate mofetil to prevent GVHD in patients with high-risk hematologic malignancies who underwent HAPLO (n = 60) or 9/10 MUD (n = 46) SCT. RESULTS: The 1-year overall and progression-free survival rates were 70% and 60%, respectively, in the HAPLO arm and 60% and 47%, respectively, in the 9/10 MUD arm. The day +100 cumulative incidence of grade II to IV acute GVHD and grade III to IV acute GVHD was 28% and 3%, respectively, in the HAPLO arm and 33% and 13%, respectively, in the 9/10 MUD arm. The 2-year cumulative incidence of chronic GVHD was 24% in the HAPLO arm and 19% in the 9/10 MUD arm. The 1-year cumulative incidence of nonrelapse mortality was 21% in the HAPLO arm and 31% in the 9/10 MUD arm, and the 1-year relapse rate was 19% in the HAPLO arm and 25% in the 9/10 MUD arm. CONCLUSIONS: Although this was a nonrandomized study and could not serve as a direct comparison between the 2 groups, the authors conclude that PTCy-based GVHD prophylaxis is effective for both HAPLO and 9/10 MUD SCTs. Prospective randomized trials will be required to compare the efficacies of alternative donor options for patients lacking HLA-matched donors. Cancer 2016;122:3316-3326. © 2016 American Cancer Society. SN - 1097-0142 UR - https://www.unboundmedicine.com/medline/citation/27404668/Results_of_a_2_arm_phase_2_clinical_trial_using_post_transplantation_cyclophosphamide_for_the_prevention_of_graft_versus_host_disease_in_haploidentical_donor_and_mismatched_unrelated_donor_hematopoietic_stem_cell_transplantation_ L2 - https://doi.org/10.1002/cncr.30180 DB - PRIME DP - Unbound Medicine ER -