Allogeneic blood or marrow transplantation with haploidentical donor and post-transplantation cyclophosphamide in patients with myelofibrosis: a multicenter study

Kunte, Siddharth; Rybicki, Lisa; Viswabandya, Auro; Tamari, Roni; Bashey, Asad; Keyzner, Alla; Iqbal, Madiha; Grunwald, Michael R.; Dholaria, Bhagirathbhai; Elmariah, Hany; Ozga, Michael; Singh, Anurag K.; Abedin, Sameem; DeZern, Amy E.; Jones, Richard J.; Gupta, Vikas; Gerds, Aaron T.; Jain, Tania

doi:10.1038/s41375-021-01449-1

Cited by 33 publications

(23 citation statements)

References 55 publications

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“…Another retrospective multicenter study of 69 patients with chronic phase MF who were treated with allogeneic blood or marrow transplantation from a haploidentical donor and received cyclophosphamide posttransplantation reported an OS of 72% (95% CI, 59%-81%), a relapse-free survival recurrence-free survival (RFS) of 44% (95% CI, 29%-59%), and a GVHD-free-RFS of 30% (95% CI, 17%-43%) at 3 years. 159 A cumulative incidence of 10% was obtained for grade 3-4 acute GVHD and 8% for extensive chronic GVHD.…”

Section: Allogeneic Hematopoietic Cell Transplantmentioning

confidence: 99%

“…A few studies have shown that larger spleen size may be associated with inferior outcomes after transplant, possibly reflecting an aggressive disease biology. [159][160][161] A spleen size $22 cm or a prior splenectomy (HR, 6.37; 95% CI, 2.02-20.1; P5.002) and bone marrow grafts (HR, 4.92; 95% CI, 1.68-14.4; P5.004) were associated with a higher incidence of relapse. 159 An univariate analysis determined that a spleen size $17 cm or a prior splenectomy was associated with worse RFS (HR, 3.50; 95% CI, 1.18-10.37; P5.02) and a higher relapse rate (subdistribution HR not calculable; P5.01).…”

Section: Allogeneic Hematopoietic Cell Transplantmentioning

confidence: 99%

“…[159][160][161] A spleen size $22 cm or a prior splenectomy (HR, 6.37; 95% CI, 2.02-20.1; P5.002) and bone marrow grafts (HR, 4.92; 95% CI, 1.68-14.4; P5.004) were associated with a higher incidence of relapse. 159 An univariate analysis determined that a spleen size $17 cm or a prior splenectomy was associated with worse RFS (HR, 3.50; 95% CI, 1.18-10.37; P5.02) and a higher relapse rate (subdistribution HR not calculable; P5.01). 160 The results of a multivariate analysis by Polverelli et al 161 demonstrated that splenectomy was associated with reduced NRM (HR, 0.64; 95% CI, 0.44-0.93; P5.018), a higher risk of relapse (HR, 1.43; 95% CI, 1.01-2.02; P5.042), but no effect on OS (HR, 0.86; 95% CI, 0.67-1.12; P5.274).…”

Section: Allogeneic Hematopoietic Cell Transplantmentioning

confidence: 99%

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Myeloproliferative Neoplasms, Version 3.2022, NCCN Clinical Practice Guidelines in Oncology

Gerds

Gotlib

Ali

et al. 2022

Journal of the National Comprehensive Cancer Network

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The classic Philadelphia chromosome–negative myeloproliferative neoplasms (MPN) consist of myelofibrosis, polycythemia vera, and essential thrombocythemia and are a heterogeneous group of clonal blood disorders characterized by an overproduction of blood cells. The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for MPN were developed as a result of meetings convened by a multidisciplinary panel with expertise in MPN, with the goal of providing recommendations for the management of MPN in adults. The Guidelines include recommendations for the diagnostic workup, risk stratification, treatment, and supportive care strategies for the management of myelofibrosis, polycythemia vera, and essential thrombocythemia. Assessment of symptoms at baseline and monitoring of symptom status during the course of treatment is recommended for all patients. This article focuses on the recommendations as outlined in the NCCN Guidelines for the diagnosis of MPN and the risk stratification, management, and supportive care relevant to MF.

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Section: Allogeneic Hematopoietic Cell Transplantmentioning

confidence: 99%

Section: Allogeneic Hematopoietic Cell Transplantmentioning

confidence: 99%

Section: Allogeneic Hematopoietic Cell Transplantmentioning

confidence: 99%

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Myeloproliferative Neoplasms, Version 3.2022, NCCN Clinical Practice Guidelines in Oncology

Gerds

Gotlib

Ali

et al. 2022

Journal of the National Comprehensive Cancer Network

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“…RRs were relatively high at 31%, however Grade 3–4 acute GVHD was reported in only 10%, and extensive chronic GVHD in 8%. 76 GF occurred in 6%, but rates of PGF were not reported. With the general shift over the last decade away from umbilical cord donor transplant, there is limited research into the efficacy of cord transplant for MF.…”

Section: How To Transplantmentioning

confidence: 96%

Innovative strategies to improve hematopoietic stem cell transplant outcomes in myelofibrosis

et al. 2022

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Myelofibrosis (MF) is a clonal myeloproliferative neoplasm characterized by inflammation, marrow fibrosis, and an inherent risk of blastic transformation. Hematopoietic allogeneic stem cell transplant is the only potentially curative therapy for this disease, however, survival gains observed for other transplant indications over the past two decades have not been realized for MF. The role of transplantation may also evolve with the use of novel targeted agents. The chronic inflammatory state associated with MF necessitates pretransplantation assessment of end-organ function. Applying the transplant methodology employed for other myeloid disorders to patients with MF fails to acknowledge differences in the underlying disease pathophysiology. Limited understanding of the causes of poor transplant outcomes in this cohort has prevented refinement of transplant eligibility criteria in MF. There is increasing evidence of heterogeneity in molecular disease grade, beyond the clinical manifestations which have traditionally guided transplant timing. Exploring the physiological consequences of disease chronicity unique to MF, acknowledging the heterogeneity in disease grade, and using advanced prognostic models, molecular diagnostics and other organ function diagnostic tools, we present an innovative review of strategies with the potential to improve transplant outcomes in this disease. Larger, prospective studies which consider the impact of molecular-based disease grade are needed for MF transplantation.

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“…We have previously reported that spleen size, (maximum spleen longitudinal size recorded on ultrasound pretransplant—with a cut off of 22 cm), and transfusion burden (with a cut off of 20 red blood cell transfusions) were predictive of outcome after HSCT [ 9 ]. A recent study in patients with myelofibrosis grafted from haploidentical donor, has confirmed a negative impact of a large spleen (>22 cm) on relapse, but not on survival [ 10 ], and splenectomy may be beneficial in patients with a very large spleen [ 11 ]. The role of transfusion burden has not been evaluated to our knowledge.…”

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confidence: 99%

Predicting the outcome for patients with myelofibrosis undergoing an allogeneic hemopoietic stem cell transplant

et al. 2022

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Allogeneic blood or marrow transplantation with haploidentical donor and post-transplantation cyclophosphamide in patients with myelofibrosis: a multicenter study

Cited by 33 publications

References 55 publications

Myeloproliferative Neoplasms, Version 3.2022, NCCN Clinical Practice Guidelines in Oncology

Myeloproliferative Neoplasms, Version 3.2022, NCCN Clinical Practice Guidelines in Oncology

Innovative strategies to improve hematopoietic stem cell transplant outcomes in myelofibrosis

Predicting the outcome for patients with myelofibrosis undergoing an allogeneic hemopoietic stem cell transplant

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