INTRODUCTION: Although a number of studies were published on the efficacy of post-transplantation cyclophosphamide (PTCy) for graft-versus-host disease (GVHD) prophylaxis, no large studies prospectively evaluated this strategy in related, unrelated, and haploidentical grafts. METHODS: In this study, GVHD prophylaxis for 57 matched bone marrow (MBM) grafts consisted of single-agent PTCy, for 88 matched PBSC grafts (MPBSC) consisted of PTCy, tacrolimus, and mycophenolate mofetil (MMF) 30 mg/kg, and for 55 mismatched grafts (MMGs) consisted of PTCy, tacrolimus and MMF 45 mg/kg. RESULTS: The study met the primary endpoint to demonstrate equivalent rates of acute GVHD grade II-IV (11%, 17%,19%, P = .46), III-IV (7%, 2%, 6%, P = .41), and moderate and severe chronic GVHD (22%, 11%, 15%, P = .23). There was also no differences in non-relapse mortality (11% vs 15% vs 17%, P = .75), overall survival (63% vs 71% vs 56%, P = .72), event-free-survival (51% vs 66% vs 48%, P = .32) for MBM, MPBSC, and MMG groups, respectively. Toxicity was comparable between groups except higher incidence of nephrotoxicity in combination arms (P = .0005) and higher incidence of graft failures in MMG group (P = .004). CONCLUSION: The suggested risk-adapted PTCy-based prophylaxis is feasible and is associated with low GVHD incidence and mortality in all types of grafts. The study was registered on clinicaltrials.gov (NCT02294552).
INTRODUCTION: Although a number of studies were published on the efficacy of post-transplantation cyclophosphamide (PTCy) for graft-versus-host disease (GVHD) prophylaxis, no large studies prospectively evaluated this strategy in related, unrelated, and haploidentical grafts. METHODS: In this study, GVHD prophylaxis for 57 matched bone marrow (MBM) grafts consisted of single-agent PTCy, for 88 matched PBSC grafts (MPBSC) consisted of PTCy, tacrolimus, and mycophenolate mofetil (MMF) 30 mg/kg, and for 55 mismatched grafts (MMGs) consisted of PTCy, tacrolimus and MMF 45 mg/kg. RESULTS: The study met the primary endpoint to demonstrate equivalent rates of acute GVHD grade II-IV (11%, 17%,19%, P = .46), III-IV (7%, 2%, 6%, P = .41), and moderate and severe chronic GVHD (22%, 11%, 15%, P = .23). There was also no differences in non-relapse mortality (11% vs 15% vs 17%, P = .75), overall survival (63% vs 71% vs 56%, P = .72), event-free-survival (51% vs 66% vs 48%, P = .32) for MBM, MPBSC, and MMG groups, respectively. Toxicity was comparable between groups except higher incidence of nephrotoxicity in combination arms (P = .0005) and higher incidence of graft failures in MMG group (P = .004). CONCLUSION: The suggested risk-adapted PTCy-based prophylaxis is feasible and is associated with low GVHD incidence and mortality in all types of grafts. The study was registered on clinicaltrials.gov (NCT02294552).
Authors: Mi Kwon; Rebeca Bailén; María Jesús Pascual-Cascón; Ana Isabel Gallardo-Morillo; Abel García Sola; Pascual Balsalobre; Laura Solán; Nieves Dorado; Cristina Muñoz; David Serrano; Carolina Martínez-Laperche; Ismael Buño; Javier Anguita; José Luis Díez-Martin Journal: Blood Adv Date: 2019-11-12
Authors: Dimana Dimitrova; Juan Gea-Banacloche; Seth M Steinberg; Jennifer L Sadler; Stephanie N Hicks; Ellen Carroll; Jennifer S Wilder; Mark Parta; Lauren Skeffington; Thomas E Hughes; Jenny E Blau; Miranda M Broadney; Jeremy J Rose; Amy P Hsu; Rochelle Fletcher; Natalia S Nunes; Xiao-Yi Yan; William G Telford; Veena Kapoor; Jeffrey I Cohen; Alexandra F Freeman; Elizabeth Garabedian; Steven M Holland; Andrea Lisco; Harry L Malech; Luigi D Notarangelo; Irini Sereti; Nirali N Shah; Gulbu Uzel; Christa S Zerbe; Daniel H Fowler; Ronald E Gress; Christopher G Kanakry; Jennifer A Kanakry Journal: Biol Blood Marrow Transplant Date: 2019-09-04 Impact factor: 5.742
Authors: Matthew J Wieduwilt; Leland Metheny; Mei-Jie Zhang; Hai-Lin Wang; Noel Estrada-Merly; David I Marks; A Samer Al-Homsi; Lori Muffly; Nelson Chao; David Rizzieri; Robert Peter Gale; Shahinaz M Gadalla; Mitchell Cairo; Alberto Mussetti; Steven Gore; Vijaya Raj Bhatt; Sagar S Patel; Fotios V Michelis; Yoshihiro Inamoto; Sherif M Badawy; Edward Copelan; Neil Palmisiano; Mohamed A Kharfan-Dabaja; Hillard M Lazarus; Siddhartha Ganguly; Christopher Bredeson; Miguel Angel Diaz Perez; Ryan Cassaday; Bipin N Savani; Karen Ballen; Rodrigo Martino; Baldeep Wirk; Ulrike Bacher; Mahmoud Aljurf; Asad Bashey; Hemant S Murthy; Jean A Yared; Ibrahim Aldoss; Nosha Farhadfar; Hongtao Liu; Hisham Abdel-Azim; Edmund K Waller; Melhem Solh; Matthew D Seftel; Marjolein van der Poel; Michael R Grunwald; Jane L Liesveld; Rammurti T Kamble; Joseph McGuirk; Reinhold Munker; Jean-Yves Cahn; Jong Wook Lee; César O Freytes; Maxwell M Krem; Lena E Winestone; Usama Gergis; Sunita Nathan; Richard F Olsson; Leo F Verdonck; Akshay Sharma; Olle Ringdén; Brian D Friend; Jan Cerny; Hannah Choe; Saurabh Chhabra; Taiga Nishihori; Sachiko Seo; Biju George; Lee Ann Baxter-Lowe; Gerhard C Hildebrandt; Marcos de Lima; Mark Litzow; Partow Kebriaei; Christopher S Hourigan; Muhammad Bilal Abid; Daniel J Weisdorf; Wael Saber Journal: Blood Adv Date: 2022-01-11