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Long-term remission of myelofibrosis following nonmyeloablative allogeneic peripheral blood progenitor cell transplantation in older age: the Scripps Clinic experience

Bone Marrow Transplantation volume 34, pages 273–274 (2004)Cite this article

An Erratum to this article was published on 15 October 2004

Myelofibrosis with myeloid metaplasia (MMM) is classified as a chronic myeloproliferative disorder and this term encompasses idiopathic myelofibrosis (IMF) as well as MMM occurring in patients with a prior history of essential thrombocythemia or polycythemia vera.1 IMF has the worst prognosis among all chronic myeloproliferative diseases (MPD) with a median survival not exceeding 5.5 years.1, 2 Studies have proven that the trilineage proliferation in IMF is of clonal hematopoietic stem-cell origin and that standard therapies are palliative in nature and do not change the natural history of the disease.1 Growing data in the literature support the notion that allogeneic transplantation offers the only potentially curative option in myelofibrosis.3, 4, 5, 6, 7 The majority of patients are diagnosed at age above 60 years and paradoxically this is the group that continues to present a therapeutic dilemma owing to increased toxicity of the conventional myeloablative preparative regimens as well as the suggestion of inferior transplant outcomes in advanced age reported in two largest studies on allogeneic stem-cell transplantation for myelofibrosis.3, 4 Nonmyeloablative conditioning thus represents a promising alternative. There are two small studies on this approach in seven patients with IMF aged 44–58 years5, 6 and no data exist to guide the therapy in a more advanced age group. We hereby describe our experience with nonmyeloablative allogeneic peripheral blood progenitor cell transplantation (PBPCT) in an elderly patient with IMF.

This elderly patient tolerated the conditioning very well with no grade 3 or 4 regimen-related toxicities. He had prompt achievement of neutrophil (day 12) and platelet (day 18) engraftment, comparing favorably with other reported reduced-intensity regimens.6, 7 Red blood cell transfusion independence was reached on day 79. Full donor chimerism was present in the bone marrow and peripheral blood on day 30. Day 100 bone marrow biopsy was consistent with resurgence of normal hematopoiesis and a 2-year bone marrow biopsy showed continued morphologic remission with findings of normal cellularity with trilineage hematopoiesis and no histopathologic features of IMF (Figure 1b). The patient's splenomegaly resolved and he remains free of disease over 3 months post transplant with minimal cGVHD involving oral mucosa that is under excellent control on low-dose cyclosporine and budesonide.

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  1. Division of Hematology and Oncology, Scripps Clinic, La Jolla, CA, USA

    N Greyz, W E Miller, J Andrey & J Mason

  2. Scripps Blood and Marrow Transplant, Scripps Clinic, La Jolla, CA, USA

    W E Miller, J Andrey & J Mason

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Greyz, N., Miller, W., Andrey, J. et al. Long-term remission of myelofibrosis following nonmyeloablative allogeneic peripheral blood progenitor cell transplantation in older age: the Scripps Clinic experience. Bone Marrow Transplant 34, 273–274 (2004). https://doi.org/10.1038/sj.bmt.1704578

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