Thiotepa-busulfan-fludarabine as a conditioning regimen for patients with myelofibrosis undergoing allogeneic hematopoietic transplantation: a single center experience

Memoli, Mara; Paviglianiti, Annalisa; Malard, Florent; Battipaglia, Giorgia; Brissot, Éolia; Médiavilla, Clémence; Bianchessi, Antonio; Banet, Anne; Wyngaert, Zoé Van de; Belhocine, Ramdane; Sestili, Simona; Lapusan, Simona; Hirsch, Pierre; Favale, Fabrizia; Boussaroque, Agathe; Bonnin, Agnès; Vekhoff, Anne; Legrand, Ollivier; Mohty, Mohamad; Duléry, Rémy

doi:10.1080/10428194.2020.1827246

Cited by 9 publications

(6 citation statements)

References 39 publications

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“…Median time to neutrophil and platelet engraftment was 15 and 21 days, respectively 53 . Another single‐centre experience constituting 29 patients with MF who received TBF conditioning prior to HSCT also showed high engraftment rates and promising PFS of 69% at three years 54 . All regimens used mainly busulfan‐based combinations, which led to comparable and acceptable engraftment rates and median time to engraftment.…”

Section: Conditioning Regimenmentioning

confidence: 98%

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Allogeneic haematopoietic cell transplantation for myelofibrosis: a real‐life perspective

et al. 2021

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Summary Myelofibrosis (MF) is a clonal stem cell neoplasm with heterogeneous clinical phenotypes and well‐established molecular drivers. Allogeneic haematopoietic stem cell transplantation (HSCT) offers an important curative treatment option for primary MF and post‐essential thrombocythaemia/polycythaemia vera MF or secondary MF. With a disease course that varies from indolent to highly progressive, we are now able to stratify risk of mortality through various tools including patient‐related clinical characteristics as well as molecular genetic profile. Owing to the high risk of mortality and morbidity associated with HSCT for patients with myelofibrosis, it is important to improve patient selection for transplant. Our primary goal is to comprehensively define our understanding of current practices including the role of Janus Kinase (JAK) inhibitors, to present the data behind transplantation before and after leukaemic transformation, and to introduce novel personalization of MF treatment with a proposed clinical‐molecular prognostic model to help elucidate a timepoint optimal for consideration of HSCT.

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Section: Conditioning Regimenmentioning

confidence: 98%

“…53 TBF conditioning prior to HSCT also showed high engraftment rates and promising PFS of 69% at three years. 54 All regimens used mainly busulfan-based combinations, which led to comparable and acceptable engraftment rates and median time to engraftment.…”

Section: Conditioning Regimenmentioning

confidence: 99%

Allogeneic haematopoietic cell transplantation for myelofibrosis: a real‐life perspective

et al. 2021

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“…Encouraging outcome has been obtained with the TBF combination in other studies, with a low incidence of relapse. 25,26 Below is a list of some retrospective and one prospective randomized study.…”

Section: Conditioning Regimensmentioning

confidence: 99%

“…In the same study, because of comparable NRM, the 5‐year disease free survival (DFS) was 63% versus 43%. Encouraging outcome has been obtained with the TBF combination in other studies, with a low incidence of relapse 25,26 . Below is a list of some retrospective and one prospective randomized study.…”

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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“…In recent years, the scientific community has shown an increasing interest in the use of double-alkylating conditioning regimen thiotepa-busulfan-fludarabine (TBF). Based on some retrospective studies, such a regimen has been documented to favorably affect transplant outcome, thanks to a faster donor engraftment and better disease control [58][59][60][61]. Prospective and randomized trials are warranted to confirm these preliminary results and to give conclusive information on the preferred conditioning regimen.…”

Section: Conditioning Regimenmentioning

confidence: 99%

How We Manage Myelofibrosis Candidates for Allogeneic Stem Cell Transplantation

Polverelli

Farina

D’Adda

et al. 2022

Cells

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Moving from indication to transplantation is a critical process in myelofibrosis. Most of guidelines specifically focus on either myelofibrosis disease or transplant procedure, and, currently, no distinct indication for the management of MF candidates to transplant is available. Nevertheless, this period of time is crucial for the transplant outcome because engraftment, non-relapse mortality, and relapse incidence are greatly dependent upon the pre-transplant management. Based on these premises, in this review, we will go through the path of identification of the MF patients suitable for a transplant, by using disease-specific prognostic scores, and the evaluation of eligibility for a transplant, based on performance, comorbidity, and other combined tools. Then, we will focus on the process of donor and conditioning regimens’ choice. The pre-transplant management of splenomegaly and constitutional symptoms, cytopenias, iron overload and transplant timing will be comprehensively discussed. The principal aim of this review is, therefore, to give a practical guidance for managing MF patients who are potential candidates for allo-HCT.

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Thiotepa-busulfan-fludarabine as a conditioning regimen for patients with myelofibrosis undergoing allogeneic hematopoietic transplantation: a single center experience

Cited by 9 publications

References 39 publications

Allogeneic haematopoietic cell transplantation for myelofibrosis: a real‐life perspective

Allogeneic haematopoietic cell transplantation for myelofibrosis: a real‐life perspective

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

How We Manage Myelofibrosis Candidates for Allogeneic Stem Cell Transplantation

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