Treatment of Polycythemia Vera With Busulfan (Myleran)

Louis, John

doi:10.1001/jama.1958.03000140042011

Cited by 16 publications

(3 citation statements)

References 1 publication

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“…High-dose BU is currently still used in many regimens for allogeneic HCT but is used to a much lesser extent when conditioning for autologous HCT. Historically, low-dose BU (2 mg to 6 mg orally daily) was used to treat myeloproliferative neoplasms, such as chronic myelogenous leukemia (CML), polycythemia vera [1,2], and essential thrombocytosis [3], because BU is cytotoxic to hematopoietic precursors and pluripotent stem cells. Now, high-dose BU-based conditioning is frequently used; however, because BU causes limited lymphotoxicity, it is unable to provide adequate immunosuppression as standalone conditioning.…”

Section: Introductionmentioning

confidence: 99%

Personalizing Busulfan-Based Conditioning: Considerations from the American Society for Blood and Marrow Transplantation Practice Guidelines Committee

Palmer

McCune

Perales

et al. 2016

Biology of Blood and Marrow Transplantation

128

187

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The Practice Guidelines Committee of the American Society of Blood or Marrow Transplantation (ASBMT) sought to develop an evidence-based review about personalizing busulfan-based conditioning. The Committee sought to grade the relevant published studies (June 1, 2008 through March 31, 2016) according to criteria set forth by the Steering Committee for Evidence Based Reviews from ASBMT. Unfortunately, the published literature was too heterogeneous and lacked adequately powered and sufficiently controlled studies for this to be feasible. Despite this observation, the continued interest in this topic led the Practice Guidelines Committee to develop a list of most frequently asked questions (FAQs) regarding personalized busulfan dosing. This "Considerations" document is a list of these FAQs and their responses, addressing topics of practical relevance to hematopoietic cell transplantation clinicians.

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Section: Introductionmentioning

confidence: 99%

Personalizing Busulfan-Based Conditioning: Considerations from the American Society for Blood and Marrow Transplantation Practice Guidelines Committee

Palmer

McCune

Perales

et al. 2016

Biology of Blood and Marrow Transplantation

128

187

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“…Busulfan (BU) has been used successfully in PV since 1958 [Louis, 1958]. In our practice, we reserve it for older (generally over 75 years) patients who are intolerant of HC and not suitable for IFN.…”

Section: Cytoreductive Therapymentioning

confidence: 99%

Experience with ruxolitinib in the treatment of polycythaemia vera

Alimam

Harrison

2017

Therapeutic Advances in Hematology

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Polycythaemia vera (PV) is a myeloproliferative neoplasm classically characterized by an erythrocytosis and is associated with a high risk of thromboembolic events, constitutional symptoms burden and risk of transformation to myelofibrosis and acute myeloid leukaemia. Therapy is directed at the haematocrit (HCT) to reduce the risk of thrombotic events and usually comprises low-dose aspirin and phlebotomy to maintain HCT at >45%. Frequently in addition, cytoreductive therapy is indicated in high-risk patients for normalizing haematological parameters to mitigate the occurrence of thromboembolic events. Unfortunately, there is no clear evidence that current therapies reduce the risk of transformation to myelofibrosis and for some a risk of a therapy related complication is unknown for example leukaemia due to hydroxycarbamide (HC). First-line therapy for treating PV remains HC or interferon, the latter most often in younger patients, especially those of childbearing age. However, therapy related intolerance or resistance is a common feature and results in limited treatment options for such patients. The discovery of the JAK2 V617F mutation and consequently targeted therapy with Janus kinase inhibitors, in particular ruxolitinib, has extended the spectrum of agents that can be used as second or third line in PV. The findings of the phase II trial RESPONSE and the preliminary data from RESPONSE 2 trial have identified a role for ruxolitinib in PV patients who are resistant or intolerant to HC. In this article, using clinical cases we demonstrate our experience with ruxolitinib highlighting the clinical benefits and limitations we encountered in clinical practice.

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“…Busulphan is an alkalating agent which has been reported to be effective in controlling blood counts in PV since the 1950's [37]. The EORTC conducted a randomised trial comparing busulphan to and 32 P. Busulphan gave significantly longer first remissions and significantly longer survival.…”

Section: Interferonmentioning

confidence: 99%

A review of the therapeutic agents used in the management of polycythaemia vera

McMullin

2007

Hematological Oncology

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The acquired clonal disorder Polycythaemia Vera leads to increased erythropoiesis, myelopoiesis and megakaryopoeisis. These anomalies result in an increased incidence of thromboembolic events, transformation to acute leukaemia and myelofibrosis. Treatments which aim to reduce the event rate may increase anaemia but may also affect the rate of complications. This paper reviews the evidence for the treatments which have been used in the management of the disorders over a 50 plus year period. Assessment of this evidence and its limitations form the basis for the current suggested management plans.

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Treatment of Polycythemia Vera With Busulfan (Myleran)

Cited by 16 publications

References 1 publication

Personalizing Busulfan-Based Conditioning: Considerations from the American Society for Blood and Marrow Transplantation Practice Guidelines Committee

Personalizing Busulfan-Based Conditioning: Considerations from the American Society for Blood and Marrow Transplantation Practice Guidelines Committee

Experience with ruxolitinib in the treatment of polycythaemia vera

A review of the therapeutic agents used in the management of polycythaemia vera

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