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Pediatric-onset mastocytosis: a long term clinical follow-up and correlation with bone marrow histopathology
Ashraf Uzzaman1, Irina Maric, Pierre Noel
1Laboratory of Allergic Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
Insights
Pediatric mastocytosis often resolves, especially when systemic disease is absent at diagnosis. This long-term study shows a highly encouraging prognosis for children managed symptomatically.
Area of Science:
- Pediatric Hematology
- Dermatology
- Oncology
Background:
- Pediatric mastocytosis typically presents as urticaria pigmentosa or diffuse cutaneous mastocytosis.
- While often resolving, aggressive early-life therapies are increasingly used.
- This study re-evaluates the long-term prognosis of 17 children with pediatric-onset mastocytosis initially reported in 1989.
Purpose of the Study:
- To assess the long-term prognosis of pediatric-onset mastocytosis.
- To determine the prognostic value of initial bone marrow biopsies.
- To evaluate disease regression in children managed symptomatically.
Main Methods:
- 15 of the original 17 patients were contacted for clinical status assessment.
- Original bone marrow specimens were re-examined and correlated with outcomes.
- Repeat bone marrow biopsies were performed on three patients with persistent disease.
Main Results:
- Complete disease regression occurred in 10 of 15 patients (67%).
- Major regression was observed in 3 patients (20%), and partial regression in 2 (13%).
- Initial bone marrow biopsies indicating no systemic disease predicted favorable regression.
Conclusions:
- Initial bone marrow biopsy findings are prognostic for pediatric mastocytosis.
- Children without evidence of systemic mastocytosis at diagnosis show favorable disease regression.
- The long-term prognosis for pediatric mastocytosis managed symptomatically is highly encouraging.
Background:
Pediatric onset mastocytosis usually presents as urticaria pigmentosa; and less often as diffuse cutaneous mastocytosis. While the literature indicates that disease often resolves, there has been a move to more aggressive therapy for mastocytosis early in life. We addressed the long term prognosis of pediatric-onset disease by examining 17 children with mastocytosis which we had reported on in 1989 [1].
Procedure:
We successfully contacted 15 of these patients and data was collected regarding their clinical status. Original bone marrow specimens were re-stained, re-examined, and correlated with disease outcome using consensus criteria. Three of five patients with persistent disease underwent repeat bone marrow biopsies.
Results:
There was complete regression of disease as defined by cutaneous findings and symptoms (clinical disease severity) in 10 of 15 patients (67%). Three patients had major (20%) and two had partial regression of disease (13%). Repeat marrow examinations on three patients with persistent disease documented systemic mastocytosis based on marrow findings in one patient who had partial regression of disease and was the only patient with initial morphologic evidence of systemic disease. Of the remaining two patients, one demonstrated partial regression and the other major regression of disease; and neither had evidence of systemic mastocytosis.
Conclusion:
This study demonstrates that initial bone marrow biopsies were prognostic in that those without evidence of systemic disease experienced disease regression; and that the long term prognosis for children managed symptomatically with mastocytosis is highly encouraging.
