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Primary thrombocythaemia treated with busulphan.

J E Van de Pette, A V Prochazka, T C Pearson

    British Journal of Haematology
    |February 1, 1986
    PubMed
    Summary
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    Busulfan effectively treats primary thrombocythaemia (PT) by reducing platelet counts and resolving vascular symptoms. While it increases myelofibrosis risk, busulfan demonstrates long-term efficacy and safety for PT management.

    Area of Science:

    • Hematology
    • Oncology
    • Pharmacology

    Background:

    • Primary thrombocythaemia (PT) is a myeloproliferative neoplasm characterized by elevated platelet counts.
    • Long-term treatment options for PT require careful consideration of efficacy and safety profiles.
    • Busulfan has been used in managing PT, but its long-term impact on survival and disease progression needs thorough evaluation.

    Purpose of the Study:

    • To assess the long-term efficacy and safety of busulfan in patients with primary thrombocythaemia.
    • To identify prognostic factors influencing survival in PT patients treated with busulfan.
    • To evaluate the risk of disease progression, including myelofibrosis and leukaemic transformation, under busulfan therapy.

    Main Methods:

    • A cohort of 37 patients with primary thrombocythaemia treated with busulfan was followed for up to 25 years.

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  • Platelet counts, clinical symptoms (vascular and haemorrhagic), and survival data were analyzed.
  • Cox regression analysis was employed to identify prognostic factors for survival.
  • Rates of disease progression to myelofibrosis and leukaemic transformation were documented.

    • Main Results:

    • Reduction of platelet count to <400 X 10(9)/l resolved vascular occlusive symptoms, though haemorrhagic symptoms often persisted.
    • Age was inversely correlated with survival, while vascular occlusive symptoms correlated with better survival.
    • Median survival on busulfan treatment was 9.8 years; overall mortality was 2.1 times higher than controls, with increased deaths from myelofibrosis.
    • Busulfan demonstrated efficacy and relative safety, with no significant increase in deaths from thrombosis or malignant diseases compared to expected rates.
    • Progression to myelofibrosis occurred in 24% of cases, and 9% developed polycythaemia.
    • Two additional cases of Philadelphia chromosome-positive thrombocythaemia were noted.

    Conclusions:

    • Busulfan is an effective and relatively safe option for the long-term management of primary thrombocythaemia.
    • Age and the presence of vascular occlusive symptoms are key prognostic indicators in PT patients treated with busulfan.
    • While busulfan therapy is associated with an increased risk of myelofibrosis, it does not significantly elevate risks for thrombosis or other malignancies.