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Primary myelofibrosis in children: report of 4 cases
1Department of Pediatrics, National Taiwan University Hospital, Taipei, R.O.C.
Insights
Childhood primary myelofibrosis is rare but aggressive. This study found pediatric myelofibrosis cases rapidly progressed to fatal outcomes, often within two years of diagnosis.
Area of Science:
- Hematology
- Pediatric Oncology
Background:
- Primary myelofibrosis is a rare myeloproliferative neoplasm.
- Childhood myelofibrosis presents unique diagnostic and clinical challenges.
Observation:
- Four pediatric cases of primary myelofibrosis were diagnosed between 1982-1985.
- Patients presented with anemia, fever, hepatosplenomegaly, and leukoerythroblastic changes with giant platelets.
Findings:
- Anemia was severe and refractory to treatment in most cases.
- Three patients developed leukemic transformation within two years, leading to rapid mortality.
- One patient died of sepsis shortly after diagnosis, highlighting aggressive disease progression.
Implications:
- Childhood myelofibrosis is an aggressive hematologic malignancy with a poor prognosis.
- Early diagnosis and understanding of disease trajectory are critical for pediatric patients.
Abstract:
From 1982 to 1985, four cases of primary myelofibrosis were diagnosed in our department. Three were boys and one was a girl. Their ages ranged from 7 months to 15 years. The diagnosis was made based on anemia, leukoerythroblastic change and presence of giant platelets in the peripheral blood, and a bone marrow biopsy showing myelofibrosis. Most of them had anemia, fever, and hepatosplenomegaly on admission. The anemia was severe and refractory to repeated transfusions and steroid therapy in 3 out of the 4 cases. Splenectomy was performed in 1 case, but without satisfactory results. The clinical course and blood pictures in one case resembled leukemia of megakaryocyte lineage (M7), but results of marker studies of the blast cells ruled out the possibility of M7. Three of them underwent leukemic transformation within 2 years and died soon after. The other one died of sepsis 2 weeks after diagnosis. Myelofibrosis in childhood occurs rarely, however, when it does, it always runs a rapid and fatal course.