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Clodronate in myelofibrosis: a case report
Paul Froom1, Irit Elmalah, Andrei Braester
1Institute of Hematology, Lady Davis Carmel Hospital, Haifa, Israel.
Insights
Oral bisphosphonate therapy, specifically clodronate, shows promise for treating agnogenic myeloid metaplasia (AMM). This treatment led to reduced bone marrow fibrosis and normalized blood counts in a patient with AMM, making them transfusion-independent.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Agnogenic myeloid metaplasia (AMM) is a myeloproliferative disorder characterized by bone marrow fibrosis and extramedullary hematopoiesis.
- Patients with AMM often develop pancytopenia, necessitating frequent blood transfusions.
- Conventional therapies like androgen and interferon have shown limited efficacy and potential adverse effects.
Observation:
- A 59-year-old male patient with documented AMM and pancytopenia required regular blood transfusions.
- Previous treatments with androgen therapy and interferon were ineffective and led to complications.
- The patient was subsequently treated with oral clodronate at a daily dose of 30 mg/kg.
Findings:
- Clodronate treatment resulted in a significant reduction in bone marrow fibrosis.
- Gradual normalization of blood counts was observed over an 8-month treatment period.
- The patient achieved transfusion independence, remaining so for over 33 months post-treatment.
Implications:
- Oral bisphosphonate therapy, exemplified by clodronate, may represent a viable therapeutic option for AMM.
- This approach could potentially mitigate bone marrow fibrosis and improve hematological parameters in AMM patients.
- Further investigation into bisphosphonate efficacy in AMM is warranted based on this case report.
Abstract:
A 59-year-old man had well-documented agnogenic myeloid metaplasia (AMM) with pancytopenia. Frequent blood transfusions were required over a 10-month period. Androgen therapy was not beneficial and treatment with interferon resulted in severe thrombocytopenia with no decrease in transfusion requirements. Treatment with clodronate at a daily oral dose of 30 mg/kg resulted in a marked decrease in bone marrow fibrosis, and gradual normalization of blood counts over an 8-month period. He has been transfusion independent for the last 33 months. We support the findings of a previous case report that oral bisphosphonate therapy may be of value in patients with AMM.