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[Myelofibrosis regressing under corticotherapy and intravenous immunoglobulins in an infant]
1Service de néonatologie et réanimation néonatale et infantile, CHD Félix-Guyon, Saint-Denis, La Réunion.
Insights
Primary myelofibrosis in children is rare and often lacks autoimmune markers. This case highlights successful treatment of pediatric primary myelofibrosis with intravenous immunoglobulin therapy, suggesting an autoimmune component.
Area of Science:
- Pediatric Hematology
- Autoimmune Diseases
- Bone Marrow Disorders
Background:
- Primary myelofibrosis is uncommon in pediatric populations.
- Association with autoimmune markers is primarily documented in adults.
Observation:
- An infant girl presented with severe anemia and neutropenia.
- Bone marrow biopsy revealed reticulinic myelofibrosis with dysgranulopoiesis.
- The condition progressed to agranulocytosis and thrombocytopenia.
Findings:
- Autoimmune etiology was suspected due to anti-granulocyte antibodies and a positive Coombs test.
- Corticosteroids showed limited efficacy, primarily on platelet counts.
- Intravenous immunoglobulin therapy proved effective in resolving cytopenias.
Implications:
- Intravenous immunoglobulin therapy may be a viable treatment for pediatric primary myelofibrosis with autoimmune features.
- This case expands the understanding of autoimmune associations in pediatric myelofibrosis.
- Further research is warranted to explore autoimmune mechanisms in childhood primary myelofibrosis.
Background:
Primary myelofibrosis is rare in infants and children; its association with auto-immune markers has only been reported in adults.
Case Report:
An 8 month-old girl was admitted because of severe anemia and neutropenia. The marrow aspirate showed dysgranulopoiesis and partial interruption of maturation after the myelocyte level. The bone marrow biopsy revealed reticulinic myelofibrosis. The condition worsened with development of agranulocytosis and thrombocytopenia. Investigations ruled out malignant hemopathy, metastatic infiltration of the marrow and osteopathy. A myelodysplastic syndrome was discussed, but presence of anti-granulocyte auto-antibodies and positive Coombs test led to consider an autoimmune etiology. A corticosteroid therapy was attempted, effective only on the platelet lineage. Addition of intravenous gammaglobulin therapy corrected the problem. After a 24 month-course of the disease, it was necessary to prolong therapy.
Conclusion:
The efficacy of gammaglobulins may be an additional argument for auto-immunity, although no other auto-immune pattern has been observed in our patient, contrary to reported cases in adults.