Paediatric primary myelofibrosis (PMF) is exceedingly rare and distinct compared with adult PMF. It is characterised by peripheral blood cytopenias, leucoerythroblastosis, reticulin fibrosis, extramedullary haematopoiesis and hepatosplenomegaly. In the absence of laid down diagnostic criteria, the diagnosis is largely of exclusion. Though early haematological stem cell transplant (HSCT) remains the treatment of choice, spontaneous remission or remission with steroids and/or cytoreductive agents is described in around 20% of cases of paediatric PMF. Moreover, HSCT in paediatric PMF is associated with high mortality (30%–45%). Therefore, it may be prudent to consider a trial of steroids and/or cytoreductive agents in all transfusion-dependent paediatric PMF while considering HSCT and ongoing bone marrow donor search. We describe one such infant with PMF who had complete remission of clinical and haematological parameters with a combination therapy of steroids and hydroxyurea.
- Haematology (drugs and medicines)
- Haematology (incl blood transfusion)
- Malignant and Benign haematology
- Medical management
- Therapeutic indications
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Contributors SK was involved with the conceptualization, diagnosis, clinical care, management and final manuscript writing. PM, KS, PT were involved in clinical care, diagnosis, management of patient. All authors were responsible for drafting of the text, sourcing and editing of clinical images, investigation results, and critical revision for important intellectual content. All authors were accountable for the content of article and ensure that all questions regarding the accuracy or integrity of the article are investigated and resolved. All authors have given final approval for article.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Case reports provide a valuable learning resource for the scientific community and can indicate areas of interest for future research. They should not be used in isolation to guide treatment choices or public health policy.
Competing interests None declared.
Provenance and peer review Not commissioned; externally peer reviewed.