TY - JOUR
T1 - Intensive pediatric chemotherapy regimen (PNET HR+5) in adult high-risk medulloblastoma and pineoblastoma patients
AU - Larrouquere, Louis
AU - Dufour, Christelle
AU - Faure-Conter, Cécile
AU - Alapetite, Claire
AU - Meyronet, David
AU - Bolle, Stéphanie
AU - Bonneville-Levard, Alice
AU - Sunyach, Marie Pierre
AU - Laurence, Valérie
AU - Frappaz, Didier
N1 - Publisher Copyright:
© The Author(s) 2024. Published by Oxford University Press, the Society for Neuro-Oncology and the European Association of Neuro-Oncology.
PY - 2024/1/1
Y1 - 2024/1/1
N2 - Background. High-risk medulloblastoma (HRMB) is rare in adults. The 5-year overall survival rate is less than 60%. We present here a retrospective analysis of adults treated with an intensive pediatric chemo-radiotherapy regimen PNET HR + 5: NCT00936156. Methods. Eighteen patients over the age of 20 (range, 20–33 years) with HRMB (n = 13), pinealoblastoma (n = 4), and central nervous system embryonal tumor (n = 1) were treated with 2 courses of carboplatin-etoposide followed by 2 courses of high-dose thiotepa (HDT) with autologous hematopoietic stem-cell rescue. A craniospinal irradiation (CSI; 36 Gy craniospinal axis then a boost of 18 Gy to the primary tumor site) was then initiated within 150 days of surgery, completed with 6 cycles of temozolomide; the axis irradiation was not mandatory for non-metastatic pinealoblastoma. Results. We observed no progression under chemotherapy and no toxic death. Four patients received only 1 HDT. Two non-metastatic pinaloblastomas received only focal irradiation. One medulloblastoma received only 25 Gy on the axis. 56% (10/18) received 6 cycles of temozolomide. No long-term toxicity was recorded. The median time between surgery and CSI was 175 days (range, 115–250). With a median follow-up of 6.0 years (range, 2.6–9), the progression-free survival and overall survival rates for medulloblastoma were respectively 65% (95% CI: 31%–86%) and 76% (95% CI: 42%–91%) at 5 years. Conclusions. The PNET HR + 5 regimen showed promising results in an adult population, with a meaningful improvement in progression-free survival and overall survival in patients with HRMB.
AB - Background. High-risk medulloblastoma (HRMB) is rare in adults. The 5-year overall survival rate is less than 60%. We present here a retrospective analysis of adults treated with an intensive pediatric chemo-radiotherapy regimen PNET HR + 5: NCT00936156. Methods. Eighteen patients over the age of 20 (range, 20–33 years) with HRMB (n = 13), pinealoblastoma (n = 4), and central nervous system embryonal tumor (n = 1) were treated with 2 courses of carboplatin-etoposide followed by 2 courses of high-dose thiotepa (HDT) with autologous hematopoietic stem-cell rescue. A craniospinal irradiation (CSI; 36 Gy craniospinal axis then a boost of 18 Gy to the primary tumor site) was then initiated within 150 days of surgery, completed with 6 cycles of temozolomide; the axis irradiation was not mandatory for non-metastatic pinealoblastoma. Results. We observed no progression under chemotherapy and no toxic death. Four patients received only 1 HDT. Two non-metastatic pinaloblastomas received only focal irradiation. One medulloblastoma received only 25 Gy on the axis. 56% (10/18) received 6 cycles of temozolomide. No long-term toxicity was recorded. The median time between surgery and CSI was 175 days (range, 115–250). With a median follow-up of 6.0 years (range, 2.6–9), the progression-free survival and overall survival rates for medulloblastoma were respectively 65% (95% CI: 31%–86%) and 76% (95% CI: 42%–91%) at 5 years. Conclusions. The PNET HR + 5 regimen showed promising results in an adult population, with a meaningful improvement in progression-free survival and overall survival in patients with HRMB.
KW - adult medulloblastoma
KW - high-dose chemotherapy
KW - high-risk medulloblastoma
KW - pinaloblastoma
KW - thiotepa
UR - http://www.scopus.com/inward/record.url?scp=85211007337&partnerID=8YFLogxK
U2 - 10.1093/noajnl/vdae141
DO - 10.1093/noajnl/vdae141
M3 - Article
AN - SCOPUS:85211007337
SN - 2632-2498
VL - 6
JO - Neuro-Oncology Advances
JF - Neuro-Oncology Advances
IS - 1
M1 - vdae141
ER -