Successful Outcome in Patients with Myelofibrosis Undergoing Allogeneic Donor Hematopoietic Cell Transplantation Using Reduced Doses of Post-Transplantation Cyclophosphamide: Challenges and Review of the Literature

García‐Cadenas, Irene; Redondo, Sara; Esquirol, Albert; Portos, JM; Novelli, Silvana; Saavedra, Silvana; Moreno, Carol; Garrido, Ana; Oñate, Guadalupe; Jordi, López; Ana-Carolina, Caballero; Miqueleiz, Sara; Arguello-Tomas, Miguel; Briones, Javier; Sierra, Jorge; Martino, Rodrigo

doi:10.1016/j.jtct.2023.04.008

Cited by 4 publications

(2 citation statements)

References 40 publications

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“…Three studies evaluated reduced doses of PTCY without the addition of ATG in recipients of MRD or MUD transplants [31][32][33]. Solterman et al conducted a retrospective analysis of PTCY 40 mg/kg/day on days + 3/+4, followed by CsA and MMF, in 22 patients who underwent 1-antigen HLA-mismatched unrelated donor transplant [31].…”

Section: Discussionmentioning

confidence: 99%

Reduced Dose of Post-transplant Cyclophosphamide with Tacrolimus for the Prevention of Graft-versus-Host Disease in HLA-Matched Donor Peripheral Blood Stem Cell Transplants: A Prospective Pilot Study

Martinez,

Juárez,

Salas

et al. 2024

Preprint

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High-dose post-transplant cyclophosphamide (PTCY) (50 mg/kg/day, days + 3/+4) is an excellent strategy to prevent GVHD. However, its use is associated with adverse outcomes such as delayed engraftment, increased risk of infection, and cardiac complications. We prospectively evaluated PTCY at a reduced dose of 40 mg/kg/day on days + 3/+4 plus tacrolimus in 22 patients undergoing HLA-matched alloHSCT. At day + 100, the cumulative incidences of grade II-IV and III-IV acute GVHD were 18.2% and 4.5%, respectively. No grade IV acute GVHD or steroid-refractory disease was observed. The cumulative incidences of all-grade and moderate-severe chronic GVHD at 1-year were 11.4% and 6.4%, respectively. No patient died from transplant-related complications. Two-year OS and RFS were 77.1% and 58.3%, respectively. Neutrophil and platelet engraftment occurred at a median of 15 (IQR 14–16) and 16 days (IQR 12–23), respectively. No patient experienced primary graft failure. The cumulative incidences of bloodstream bacterial infections, polyomavirus BK hemorrhagic cystitis, HHV6 reactivation, CMV reactivation, and fungal infections were 13.6%, 9.1%, 9.1%, 4.6%, and 6%, respectively. Only one early cardiac event was observed. These results suggest that PTCY 40 mg/kg/day on a + 3/+4 schedule provides adequate immunosuppression to allow for engraftment and prevent clinically significant GVHD with a low toxicity profile.

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Section: Discussionmentioning

confidence: 99%

Reduced Dose of Post-transplant Cyclophosphamide with Tacrolimus for the Prevention of Graft-versus-Host Disease in HLA-Matched Donor Peripheral Blood Stem Cell Transplants: A Prospective Pilot Study

Martinez,

Juárez,

Salas

et al. 2024

Preprint

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“…In patients with myelofibrosis, a PTCY‐based prophylaxis has been reported with haploidentical grafts, and more recently also for HLA‐matched grafts: in the latter case GvHD is expected to be controlled better, although perhaps at the expense of a reduced graft versus leukemia (GvL), resulting in comparable overall survival of the haploidentical and HLA‐matched grafts 35,36,39 . A reduced dose of PTCY may also be used, as suggested by a recent report, with very encouraging early results, and a NRM of 7% 41 . Ex vivo T‐cell depletion is an alternative way of preventing GvHD: the Memorial Sloan Kettering has reported very interesting outcome using CD34 selection as a stem cell source, following a busulfan, melphalan fludarabine conditioning, and ATG to prevent rejection: there was no graft failure in 27 patients, with a low rate of acute and chronic GvHD and a 3 year survival of 88% 42 .…”

Section: Introductionmentioning

confidence: 99%

2024 update on allogeneic hematopoietic stem cell transplant for myelofibrosis: A review of current data and applications on risk stratification and management

Ali,

Bacigalupo

2024

American J Hematol

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BackgroundAllogeneic hemopoietic stem cell transplantation (HSCT) currently remains the only curative treatment for patients with myelofibrosis (MF). Transplant related mortality (TRM) and relapse, remain two significant complications which need to be addressed.AimsThe aim of this manuscript is to review current available reports on changes which have recently occurred, to improve the outcome of MF patients undergoing an allogeneic HSCT.MethodsPublished papers were used to analyze different aspects of allogeneic HSCT.ResultsChanges and updates are provided on selection of patients, prognostic systems, managing splenomegaly, conditioning regimens, predicting transplant outcome, stem cell sources, stem cell donors, graft versus host disease (GvHD) prophylaxis, patients with blast phase, hematopoietic reconstitution, disease markers, donor chimerism, and treatment of relapse.ConclusionsThe review outlines new transplant platforms which are now available for patients with myelofibrosis, together with persisting problems, among which, older age combined with marrow fibrosis and an inflammatory disease. Relapse also requires aggressive monitoring of drivers mutations, and early cellular therapy.

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Post-transplant cyclophosphamide compared to sirolimus/tacrolimus in reduced intensity conditioning transplants for patients with lymphoid malignancies

Fox,

García-Cadenas,

Navarro

et al. 2024

Bone Marrow Transplant

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Successful Outcome in Patients with Myelofibrosis Undergoing Allogeneic Donor Hematopoietic Cell Transplantation Using Reduced Doses of Post-Transplantation Cyclophosphamide: Challenges and Review of the Literature

Cited by 4 publications

References 40 publications

Reduced Dose of Post-transplant Cyclophosphamide with Tacrolimus for the Prevention of Graft-versus-Host Disease in HLA-Matched Donor Peripheral Blood Stem Cell Transplants: A Prospective Pilot Study

Reduced Dose of Post-transplant Cyclophosphamide with Tacrolimus for the Prevention of Graft-versus-Host Disease in HLA-Matched Donor Peripheral Blood Stem Cell Transplants: A Prospective Pilot Study

2024 update on allogeneic hematopoietic stem cell transplant for myelofibrosis: A review of current data and applications on risk stratification and management

Post-transplant cyclophosphamide compared to sirolimus/tacrolimus in reduced intensity conditioning transplants for patients with lymphoid malignancies

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