Hepatopathy-thrombocytopenia syndrome and disease relapse in pediatric wilms tumor: A 20-year retrospective analysis in a single center.

Background: Wilms tumor (WT) is the most common renal malignancy in children. With current treatments, the 5-year overall survival (OS) rate for children with WT is approximately 90 %. However, nearly 15 % of patients experience disease recurrence. A rare but severe treatment-related complication is hepatopathy-thrombocytopenia syndrome (HTS). In this study, we evaluated the characteristics, treatment outcomes, and complications of patients with WT, particularly focusing on HTS and relapse cases.

Methods: We conducted a retrospective observational study of WT cases from 2001 to 2021 at a tertiary care hospital in central Taiwan. Patient records were examined to collect information on age at diagnosis, sex, disease stage, treatment, tumor characteristics, treatment complications, and outcomes.

Results: We included 23 pediatric patients with untreated WT. Their median follow-up duration was 102 months. Their median age was 3.4 (0.8-10.8) years. The most common tumor stage was Stage II (9/23, 39.1 %), followed by Stage I (7/23, 30.4 %), III (5/23, 21.7 %), IV (1/23, 4.3 %), and V (1/23, 4.3 %). One patient with Stage IV disease had distant metastasis of the right atrium. Two patients with Stage III disease developed HTS during treatment. Relapse occurred in 3 (13 %) of 23 patients, and the average time to relapse from the initial diagnosis was 25 (range, 9-44) months. All patients survived during the follow-up period, but one continued to experience refractory lung recurrence. Two patients underwent hematopoietic stem cell transplantation for relapse/refractory disease. No disease-related mortality was noted. The 5-year event-free survival and OS rates were 86.2 % and 100 %, respectively.

Conclusion: Although WT generally has a favorable prognosis, physicians should remain mindful of potential treatment-associated complications and the risk of relapse when managing WT in children.