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Published on: March 17, 2020
Pentostatin for the treatment of chronic graft-versus-host disease in children
Jenna D Goldberg1, David A Jacobsohn, Jeffrey Margolis
1Division of Hematological Malignancies, Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, Maryland 21231, U.S.A.
Insights
Pentostatin shows promise for treating pediatric chronic graft-versus-host disease (cGVHD). Early results indicate significant symptom improvement with no increased infection risk, suggesting potential as a steroid-sparing alternative.
Area of Science:
- Hematology
- Immunology
- Pediatric Oncology
Background:
- Allogeneic stem cell transplantation is crucial for treating various hematologic malignancies.
- Chronic graft-versus-host disease (cGVHD) remains a significant complication, impacting transplant success and patient quality of life.
- Current cGVHD management often relies on high-dose corticosteroids, which carry substantial toxicity.
Observation:
- Pentostatin, a purine analog, has demonstrated efficacy in other hematologic conditions.
- Limited data exists on pentostatin's use in pediatric cGVHD.
- This study presents the initial experience of using pentostatin for refractory cGVHD in pediatric patients.
Findings:
- All five pediatric patients with refractory cGVHD experienced significant symptom improvement.
- Skin and oral manifestations of cGVHD showed notable amelioration in every patient.
- No increase in infection rates was observed during pentostatin treatment.
- No patients required permanent discontinuation of pentostatin due to adverse events.
Implications:
- Pentostatin demonstrates a favorable safety and efficacy profile in this small cohort of pediatric cGVHD patients.
- These findings support further investigation of pentostatin for cGVHD treatment.
- Consideration of pentostatin as a first-line therapy could reduce reliance on high-dose steroids, thereby mitigating treatment-related toxicities.
Abstract:
Chronic graft-versus-host disease (cGVHD) is a major barrier to successful allogeneic stem cell transplantation. Pentostatin has been used to treat cGVHD in a small series of pediatric patients. The authors report the results of the first five pediatric patients receiving pentostatin for refractory cGVHD at Johns Hopkins Hospital. In this early experience, the authors saw considerable symptom response in their patients. Every patient in this series demonstrated a significant improvement in skin and oral symptoms. An increased incidence of infection secondary to pentostatin was not observed. No patient was permanently discontinued from pentostatin subsequent to side effects. If these promising results continue, a trial of pentostatin as a first-line therapy for cGVHD should be considered to potentially reduce our dependence on high-dose steroids for its treatment.
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