Improved prognosis in disseminated histiocytosis
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Updated: Aug 18, 2026
Ex Vivo Treatment Response of Primary Tumors and/or Associated Metastases for Preclinical and Clinical Development of Therapeutics
Published on: October 2, 2014
Insights
Modern chemotherapy has significantly improved outcomes for children with disseminated histiocytosis. Clinical staging aids in predicting treatment response and prognosis for these young patients.
Area of Science:
- Pediatric Oncology
- Hematology
Background:
- Disseminated histiocytosis historically had a poor prognosis in children.
- Advances in chemotherapy have led to improved treatment outcomes.
Purpose of the Study:
- To evaluate the effectiveness of modern chemotherapy in treating disseminated histiocytosis in children.
- To assess the value of clinical and histologic staging in predicting prognosis.
Main Methods:
- Fourteen children with histiocytosis were clinically staged (Stage I: no organ dysfunction; Stage II: organ dysfunction).
- Histologic staging (Benign/Malignant) was also performed.
- Treatment involved oral chlorambucil or combination chemotherapy (vinblastine and other agents).
Main Results:
- Thirteen of 14 children responded to treatment and are alive.
- Clinical staging was valuable for predicting treatment response and prognosis.
- Relapses occurred in two children on treatment and two after stopping treatment; both groups responded to re-initiated or modified therapy.
Conclusions:
- Modern chemotherapy offers a favorable prognosis for children with disseminated histiocytosis.
- Clinical staging is a useful tool in managing these patients.
- Relapsed disease can be effectively managed with adjustments in therapy.
Abstract:
The prognosis for children with disseminated histiocytosis, previously considered poor, has improved dramatically with the application of modern principles of chemotherapy. Fourteen children with histiocytosis were staged clinically as follows: those without organ dysfunction, stage I; those with organ dysfunction, stage II; and histologically (B, benign and M, malignant). They were treated with either oral chlorambucil or combination chemotherapy with vinblastine and other agents. Clinical staging was of value in predicting response to treatment and prognosis, while histologic staging was of less value. Thirteen of the 14 children responded to treatment and are alive 4 to 67 months (median 12 months) after diagnosis. Two of these relapsed on treatment, and they have responded to a change in therapy. Two children relapsed after stopping treatment and were reinduced with reintroduction of similar therapy. Initial response to treatment suggests a favourable outcome, for children who initially responded to treatment but relapsed subsequently responded to either reintroduction of the same treatment or a change in treatment.
