Long-term low-dose sirolimus therapy and successful discontinuation in an adult with kaposiform lymphangiomatosis and disseminated intravascular coagulation: a case report - Report - 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
Clinical Report: Successful Long-Term Management of Kaposiform Lymphangiomatosis
Overview
This case study reports the successful long-term management of a 23-year-old male with Kaposiform lymphangiomatosis (KLA) and disseminated intravascular coagulation (DIC) using low-dose sirolimus. After four years of treatment, the patient achieved significant clinical improvements and remained stable for five years post-discontinuation.
Background
Kaposiform lymphangiomatosis is a rare and aggressive lymphatic anomaly that often leads to severe complications, including disseminated intravascular coagulation. The disease primarily affects children but can also manifest in adults, necessitating effective treatment strategies. Current conventional therapies have limited efficacy, highlighting the need for alternative options such as sirolimus.
Data Highlights
Significant clinical, laboratory, and radiologic improvements were observed over four years of therapy with low-dose sirolimus (1 mg/day). Following discontinuation, the patient remained stable throughout five years of follow-up.
Key Findings
The patient had a confirmed NRAS p.Q61R mutation associated with KLA.
Sirolimus therapy initiated at 1 mg/day led to normalization of laboratory markers of DIC and regression of lesions.
Long-term treatment resulted in significant clinical improvements, including resolution of recurrent diarrhea and normalization of blood counts.
Discontinuation of sirolimus was followed by stable disease for five years, suggesting the feasibility of monitored treatment withdrawal.
Low-dose sirolimus was well tolerated, with only mild side effects reported.
Clinical Implications
This case underscores the potential of low-dose sirolimus as an effective long-term treatment for KLA, providing a viable option for disease management. Clinicians should consider individualized dosing and careful monitoring when planning treatment withdrawal.
Conclusion
The successful management of KLA with low-dose sirolimus in this case highlights the importance of tailored therapeutic approaches and the possibility of safe treatment discontinuation. Further studies are warranted to establish standardized protocols for similar cases.
