Long-term low-dose sirolimus therapy and successful discontinuation in an adult with kaposiform lymphangiomatosis and disseminated intravascular coagulation: a case report - Summary - MDSpire
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Long-term low-dose sirolimus therapy and successful discontinuation in an adult with kaposiform lymphangiomatosis and disseminated intravascular coagulation: a case report
To report the successful long-term management of a patient with Kaposiform lymphangiomatosis (KLA) and disseminated intravascular coagulation (DIC) using low-dose sirolimus, and to evaluate the feasibility of treatment discontinuation based on clinical outcomes.
Key Findings:
Significant clinical, laboratory, and radiologic improvements were observed during sirolimus therapy, including normalization of DIC markers.
The patient achieved durable remission and remained relapse-free for five years after discontinuation of sirolimus.
Low-dose sirolimus was well tolerated with only mild side effects, including oral ulcers.
Interpretation:
This case suggests that long-term low-dose sirolimus can effectively control disease progression in KLA and that monitored treatment withdrawal may be feasible.
Limitations:
This is a single case study, limiting generalizability.
Long-term effects and optimal discontinuation protocols for sirolimus in KLA remain unclear, and further studies are needed to assess outcomes in a larger cohort.
Conclusion:
The successful management of this case highlights the potential for individualized treatment strategies in KLA.
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This twice-monthly newsletter highlights recently published research where Dana-Farber faculty are listed as first or senior authors. The information is pulled from PubMed and this issue notes papers published from April 16 - 30.
