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Thiotepa–melphalan myeloablative therapy for high‐risk neuroblastoma
Author(s) -
Yamazaki Fumito,
Yamasaki Kai,
Kiyotani Chikako,
Hashii Yoshiko,
Shioda Yoko,
Hara Junichi,
Matsumoto Kimikazu
Publication year - 2021
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.28896
Subject(s) - thiotepa , medicine , melphalan , neuroblastoma , regimen , hazard ratio , oncology , surgery , chemotherapy , confidence interval , cyclophosphamide , biology , genetics , cell culture
Background Appropriate high‐dose chemotherapy (HDC) for high‐risk neuroblastoma has not yet been established. In Japan, a unique HDC regimen that comprises two cycles of a total of 800 mg/m 2 of thiotepa and a total of 280 mg/m 2 of melphalan is widely utilized. Methods To evaluate the safety and efficacy of this thiotepa–melphalan high‐dose therapy for high‐risk neuroblastoma, we reviewed the medical records of 41 patients with high‐risk neuroblastoma who underwent this regimen followed by autologous peripheral blood stem cell rescue between 2002 and 2012. MYCN ‐amplified high‐risk neuroblastomas were observed in 23 patients. All patients underwent intensive multidrug induction chemotherapy, but none underwent anti‐GD2 antibody immunotherapy. The primary tumor was resected at the adequate time point. Results The median follow‐up duration for living patients was 9.2 years (range 5.5–14.0 years). The 5‐year event‐free survival (EFS) and overall survival from treatment initiation were 41.5 ± 7.7% and 56.1 ± 7.8%, respectively. The 5‐year EFS of MYCN ‐amplified high‐risk neuroblastoma patients was 60.9 ± 10.2%, which was significantly superior compared with those with MYCN ‐nonamplified high‐risk neuroblastoma (16.7 ± 8.8%; p < .001). MYCN amplification was the most favorable prognostic factor for EFS (hazard ratio = 0.29; 95% confidence interval = 0.12–0.66). Of the 41 patients, three died because of regimen‐related toxicity (infection, n = 2; microangiopathy, n = 1). Conclusion The thiotepa–melphalan high‐dose therapy with thiotepa and melphalan may be effective for high‐risk neuroblastoma. However, this regimen is toxic and warrants special attention in clinical practice.