Excellent outcome of young children with nodular desmoplastic medulloblastoma treated on "Head Start" III: a multi-institutional, prospective clinical trial

被引：57

作者：

Dhall, Girish ^{[1
]}

O'Neil, Sharon H. ^{[2
,3
]}

Ji, Lingyun ^{[4
]}

Haley, Kelley ^{[5
]}

Whitaker, Ashley M. ^{[5
]}

Nelson, Marvin D. ^{[6
]}

Gilles, Floyd ^{[7
]}

Gardner, Sharon L. ^{[8
]}

Allen, Jeffrey C. ^{[8
]}

Cornelius, Albert S. ^{[9
]}

Pradhan, Kamnesh ^{[10
]}

Garvin, James H. ^{[11
]}

Olshefski, Randal S. ^{[12
]}

Hukin, Juliette ^{[13
]}

Comito, Melanie ^{[14
]}

Goldman, Stewart ^{[15
]}

Atlas, Mark P. ^{[16
]}

Walter, Andrew W. ^{[17
]}

Sands, Stephen ^{[18
]}

Sposto, Richard ^{[5
]}

Finlay, Jonathan L. ^{[12
]}

机构：

[1] Univ Alabama Birmingham, Div Pediat Hematol Oncol, Birmingham, AL USA

[2] Childrens Hosp Los Angeles CHLA, Div Neurol, Los Angeles, CA USA

[3] Childrens Hosp Los Angeles CHLA, Saban Res Inst, Los Angeles, CA USA

[4] Univ Southern Calif, Dept Prevent Med, Keck Sch Med, Los Angeles, CA 90007 USA

[5] CHLA, Div Hematol Oncol, Los Angeles, CA USA

[6] CHLA, Dept Radiol, Los Angeles, CA USA

[7] CHLA, Dept Pathol, Los Angeles, CA USA

[8] NYU Med Ctr, Div Pediat Hematol Oncol, New York, NY 10016 USA

[9] Helen DeVos Childrens Hosp, Div Pediat Hematol Oncol, Grand Rapids, MI USA

[10] Riley Hosp Children, Div Pediat Hematol Oncol, Indianapolis, IN USA

[11] New York Presbyterian Hosp, Div Pediat Hematol Oncol, New York, NY USA

[12] Nationwide Childrens Hosp, Div Pediat Hematol Oncol, Columbus, OH USA

[13] British Columbia Childrens Hosp, Div Pediat Hematol Oncol, Vancouver, BC, Canada

[14] Penn State Hershey Childrens Hosp, Div Pediat Hematol Oncol, Hershey, PA USA

[15] Lurie Childrens Hosp, Div Pediat Hematol Oncol, Chicago, IL USA

[16] Childrens Med Ctr New York, Div Pediat Hematol Oncol, New York, NY USA

[17] Alfred I duPont Hosp Children, Div Pediat Hematol Oncol, Delaware, OH USA

[18] Mem Sloan Kettering Canc Ctr, Dept Psychiat & Behav Sci, 1275 York Ave, New York, NY 10021 USA

来源：

NEURO-ONCOLOGY | 2020年 / 22卷 / 12期

关键词：

desmoplastic; infants; medulloblastoma; myeloablative chemotherapy; MALIGNANT BRAIN-TUMORS; HIGH-DOSE METHOTREXATE; LONG-TERM SURVIVORS; THAN; YEARS; CHILDHOOD MEDULLOBLASTOMA; INTENSIVE CHEMOTHERAPY; CNS TUMORS; AGE; RADIATION; SYSTEM;

D O I：

10.1093/neuonc/noaa102

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Background. "Head Start" III, was a prospective clinical trial using intensive induction followed by myeloablative chemotherapy and autologous hematopoietic cell rescue (AuHCR) to either avoid or reduce the dose/volume of irradiation in young children with medulloblastoma. Methods. Following surgery, patients received 5 cycles of induction followed by myeloablative chemotherapy using carboplatin, thiotepa, and etoposide with AuHCR. Irradiation was reserved for children >6 years old at diagnosis or with residual tumor post-induction. Results. Between 2003 and 2009, 92 children <10 years old with medulloblastoma were enrolled. Five-year eventfree survival (EFS) and overall survival (OS) rates (+/- SE) were 46 +/- 5% and 62 +/- 5% for all patients, 61 +/- 8% and 77 +/- 7% for localized medulloblastoma, and 35 +/- 7% and 52 +/- 7% for disseminated patients. Nodular/desmoplastic (ND) medulloblastoma patients had 5-year EFS and OS (+/- SE) rates of 89 +/- 6% and 89 +/- 6% compared with 26 +/- 6% and 53 +/- 7% for classic and 38 +/- 13% and 46 +/- 14% for large-cell/anaplastic (LCA) medulloblastoma, respectively. In multivariate Cox regression analysis, histology was the only significant independent predictor of EFS after adjusting for stage, extent of resection, regimen, age, and sex (P <0.0001). Five-year irradiation-free EFS was 78 +/- 8% for ND and 21 +/- 5% for classic/LCA medulloblastoma patients. Myelosuppression was the most common toxicity, with 2 toxic deaths. Twenty-four survivors completed neurocognitive evaluation at a mean of 4.9 years post-diagnosis. IQ and memory scores were within average range overall, whereas processing speed and adaptive functioning were low-average. Conclusion. We report excellent survival and preservation of mean IQ and memory for young children with ND medulloblastoma using high-dose chemotherapy, with most patients surviving without irradiation.

引用

页码：1862 / 1872

页数：11

共 43 条

[1]

[Anonymous], 2012, WECHSLER PRESCHOOL P

[2]

[Anonymous], 2021, IBM SPSS Statistics for Mac (Version 28.0) [Computer software]

[3]

[Anonymous], 2016, Wechsler Intelligence Scale for ChildrenFifth Edition: Australian and New Zealand Standardised Edition. (WISCV)

[4]

Ashley DM, 2012, J CLIN ONCOL, V19, P3470

[5] Intertumoral Heterogeneity within Medulloblastoma Subgroups [J].

Cavalli, Florence M. G. ;

Remke, Marc ;

Rampasek, Ladislav ;

Peacock, John ;

Shih, David J. H. ;

Luu, Betty ;

Garzia, Livia ;

Torchia, Jonathon ;

Nor, Carolina ;

Morrissy, A. Sorana ;

Agnihotri, Sameer ;

Thompson, Yuan Yao ;

Kuzan-Fischer, Claudia M. ;

Farooq, Hamza ;

Isaev, Keren ;

Daniels, Craig ;

Cho, Byung-Kyu ;

Kim, Seung-Ki ;

Wang, Kyu-Chang ;

Lee, Ji Yeoun ;

Grajkowska, Wieslawa A. ;

Perek-Polnik, Marta ;

Vasiljevic, Alexandre ;

Faure-Conter, Cecile ;

Jouvet, Anne ;

Giannini, Caterina ;

Rao, Amulya A. Nageswara ;

Li, Kay Ka Wai ;

Ng, Ho-Keung ;

Eberhart, Charles G. ;

Pollack, Ian F. ;

Hamilton, Ronald L. ;

Gillespie, G. Yancey ;

Olson, James M. ;

Leary, Sarah ;

Weiss, William A. ;

Lach, Boleslaw ;

Chambless, Lola B. ;

Thompson, Reid C. ;

Cooper, Michael K. ;

Vibhakar, Rajeev ;

Hauser, Peter ;

van Veelen, Marie-Lise C. ;

Kros, Johan M. ;

French, Pim J. ;

Ra, Young Shin ;

Kumabe, Toshihiro ;

Lopez-Aguilar, Enrique ;

Zitterbart, Karel ;

Sterba, Jaroslav .

CANCER CELL, 2017, 31 (06) :737-+

[6] AN OPERATIVE STAGING SYSTEM AND A MEGAVOLTAGE RADIOTHERAPEUTIC TECHNIC FOR CEREBELLAR MEDULLOBLASTOMAS [J].

CHANG, CH ;

HOUSEPIAN, EM ;

HERBERT, C .

RADIOLOGY, 1969, 93 (06) :1351-+

[7] NEUROBEHAVIORAL AND NEUROLOGIC OUTCOME IN LONG-TERM SURVIVORS OF POSTERIOR-FOSSA BRAIN-TUMORS - ROLE OF AGE AND PERIOPERATIVE FACTORS [J].

CHAPMAN, CA ;

WABER, DP ;

BERNSTEIN, JH ;

POMEROY, SL ;

LAVALLY, B ;

SALLAN, SE ;

TARBELL, N .

JOURNAL OF CHILD NEUROLOGY, 1995, 10 (03) :209-212

[8] Feasibility and response to induction chemotherapy intensified with high-dose methotrexate for young children with newly diagnosed high-risk disseminated medulloblastoma [J].

Chi, SN ;

Gardner, SL ;

Levy, AS ;

Knapp, EA ;

Miller, DC ;

Wisoff, JH ;

Weiner, HL ;

Finlay, JL .

JOURNAL OF CLINICAL ONCOLOGY, 2004, 22 (24) :4881-4887

[9] Pilot Study of Intensive Chemotherapy With Peripheral Hematopoietic Cell Support for Children Less Than 3 Years of Age With Malignant Brain Tumors, the CCG-99703 Phase I/II Study. A Report From the Children's Oncology Group [J].

Cohen, Bruce H. ;

Geyer, J. Russell ;

Miller, Douglas C. ;

Curran, John G. ;

Zhou, Tianni ;

Holmes, Emi ;

Ingles, Sue Ann ;

Dunkel, Ira J. ;

Hilden, Joanne ;

Packer, Roger J. ;

Pollack, Ian F. ;

Gajjar, Amar ;

Finlay, Jonathan L. .

PEDIATRIC NEUROLOGY, 2015, 53 (01) :31-46

[10]

Cohen M., 1994, CHILDRENS MEMORY SCA

← 1 2 3 4 5 →