1. Promising survival rate but high incidence of treatment‐related mortality after reduced‐dose craniospinal radiotherapy and tandem high‐dose chemotherapy in patients with high‐risk medulloblastoma
- Author
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Ji Won Lee, Do Hoon Lim, Ju Kyung Hyun, Keon Hee Yoo, Ki Woong Sung, Hee Won Cho, Yoo-Sook Joung, Hee Young Ju, Hong Hoe Koo, Hyung Jin Shin, and Yeon-Lim Suh
- Subjects
0301 basic medicine ,Oncology ,Male ,Cancer Research ,medicine.medical_treatment ,0302 clinical medicine ,Craniospinal Irradiation ,Antineoplastic Combined Chemotherapy Protocols ,Cumulative incidence ,Child ,Original Research ,Incidence ,Radiotherapy Dosage ,Induction Chemotherapy ,lcsh:Neoplasms. Tumors. Oncology. Including cancer and carcinogens ,Progression-Free Survival ,Survival Rate ,Chemotherapy, Adjuvant ,030220 oncology & carcinogenesis ,Child, Preschool ,high‐dose chemotherapy ,Disease Progression ,Female ,craniospinal radiotherapy ,Adult ,medicine.medical_specialty ,Adolescent ,medulloblastoma ,lcsh:RC254-282 ,03 medical and health sciences ,Young Adult ,Internal medicine ,medicine ,Humans ,Radiology, Nuclear Medicine and imaging ,Cerebellar Neoplasms ,Survival rate ,Medulloblastoma ,Chemotherapy ,business.industry ,Induction chemotherapy ,Clinical Cancer Research ,medicine.disease ,Radiation therapy ,Regimen ,treatment‐related mortality ,030104 developmental biology ,long‐term follow‐up ,Neoplasm Recurrence, Local ,business ,Dyslipidemia ,Follow-Up Studies - Abstract
Background In this study, we report the follow‐up results of reduced dose of craniospinal radiotherapy (CSRT) followed by tandem high‐dose chemotherapy (HDCT) in patients with high‐risk medulloblastoma (MB). Methods Newly diagnosed high‐risk MB patients (metastatic disease, postoperative residual tumor >1.5 cm2, or large cell/anaplastic histology) over 3 years of age were enrolled in this study. Two cycles of pre‐RT chemotherapy, radiotherapy (RT) including reduced‐dose CSRT (23.4 or 30.6 Gy), four cycles of post‐RT chemotherapy, and tandem HDCT were administered. NanoString and DNA sequencing were performed using archival tissues. Results In all, 40 patients were enrolled, and molecular subgrouping was possible in 21 patients (2 wingless, 3 sonic hedgehog, 8 Group 3, and 8 group 4). All patients including two patients who experienced progression during the induction chemotherapy underwent HDCT. Relapse/progression occurred only in four patients (5‐year cumulative incidence [CI] 10.4 ± 0.3%). However, six patients died from treatment‐related mortality (TRM) (four acute TRMs and two late TRMs) resulting in 18.5 ± 0.5% of 5‐year CI. Taken together, the 5‐year event‐free survival and overall survival were 71.1 ± 8.0% and 73.2 ± 7.9%, respectively. Late effects were evaluated in 25 patients and high‐tone hearing loss, endocrine dysfunction, dyslipidemia, and growth retardation were common. Conclusions The strategy using tandem HDCT following reduced‐dose CSRT showed promising results in terms of low relapse/progression rate; however, the high TRM rate indicates that modification of HDCT regimen and careful selection of patients who can benefit from HDCT will be needed in the future study., Tandem high‐dose chemotherapy (HDCT) after reduced‐dose craniospinal radiotherapy resulted in a low progression rate in high‐risk medulloblastoma. However, the high treatment‐related mortality rate indicates the need to modify the HDCT regimen and carefully select patients who may benefit from HDCT.
- Published
- 2020