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Excellent outcome of young children with nodular desmoplastic medulloblastoma treated on "Head Start" III: a multi-institutional, prospective clinical trial.
Dhall, Girish; O'Neil, Sharon H; Ji, Lingyun; Haley, Kelley; Whitaker, Ashley M; Nelson, Marvin D; Gilles, Floyd; Gardner, Sharon L; Allen, Jeffrey C; Cornelius, Albert S; Pradhan, Kamnesh; Garvin, James H; Olshefski, Randal S; Hukin, Juliette; Comito, Melanie; Goldman, Stewart; Atlas, Mark P; Walter, Andrew W; Sands, Stephen; Sposto, Richard; Finlay, Jonathan L.
Affiliation
  • Dhall G; Division of Pediatric Hematology-Oncology, University of Alabama Birmingham, Birmingham, Alabama, USA.
  • O'Neil SH; Division of Neurology and The Saban Research Institute, Children's Hospital Los Angeles (CHLA), Los Angeles, California, USA.
  • Ji L; Department of Preventive Medicine, Keck School of Medicine at the University of Southern California, Los Angeles, California, USA.
  • Haley K; Division of Hematology-Oncology CHLA, Los Angeles, California, USA.
  • Whitaker AM; Division of Hematology-Oncology CHLA, Los Angeles, California, USA.
  • Nelson MD; Department of Radiology CHLA, Los Angeles, California, USA.
  • Gilles F; Department of Pathology CHLA, Los Angeles, California, USA.
  • Gardner SL; Division of Pediatric Hematology-Oncology, NYU Medical Center, New York, New York, USA.
  • Allen JC; Division of Pediatric Hematology-Oncology, NYU Medical Center, New York, New York, USA.
  • Cornelius AS; Division of Pediatric Hematology-Oncology, Helen DeVos Children's Hospital, Grand Rapids, Michigan, USA.
  • Pradhan K; Division of Pediatric Hematology-Oncology, Riley Hospital for Children, Indianapolis, Indiana, USA.
  • Garvin JH; Division of Pediatric Hematology-Oncology, New York Presbyterian Hospital, New York, New York, USA.
  • Olshefski RS; Division of Pediatric Hematology-Oncology, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Hukin J; Division of Pediatric Hematology-Oncology, British Columbia Children's Hospital, Vancouver, British Columbia, Canada.
  • Comito M; Division of Pediatric Hematology-Oncology, Penn State Hershey Children's Hospital, Hershey, Pennsylvania, USA.
  • Goldman S; Division of Pediatric Hematology-Oncology, Lurie Children's Hospital, Chicago, Illinois, USA.
  • Atlas MP; Division of Pediatric Hematology-Oncology, Children's Medical Center of New York, New York, New York, USA.
  • Walter AW; Division of Pediatric Hematology-Oncology, Alfred I. duPont Hospital for Children, Wilmington, Delaware, USA.
  • Sands S; Departments of Psychiatry and Behavioral Sciences and Pediatrics, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
  • Sposto R; Division of Hematology-Oncology CHLA, Los Angeles, California, USA.
  • Finlay JL; Division of Pediatric Hematology-Oncology, Nationwide Children's Hospital, Columbus, Ohio, USA.
Neuro Oncol ; 22(12): 1862-1872, 2020 12 18.
Article in En | MEDLINE | ID: mdl-32304218
ABSTRACT

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 event-free survival (EFS) and overall survival (OS) rates (±SE) were 46 ±â€…5% and 62 ±â€…5% for all patients, 61 ±â€…8% and 77 ±â€…7% for localized medulloblastoma, and 35 ±â€…7% and 52 ±â€…7% for disseminated patients. Nodular/desmoplastic (ND) medulloblastoma patients had 5-year EFS and OS (±SE) rates of 89 ±â€…6% and 89 ±â€…6% compared with 26 ±â€…6% and 53 ±â€…7% for classic and 38 ±â€…13% and 46 ±â€…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 ±â€…8% for ND and 21 ±â€…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.
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Cerebellar Neoplasms / Early Intervention, Educational / Medulloblastoma Type of study: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Limits: Child / Child, preschool / Female / Humans / Male Language: En Journal: Neuro Oncol Journal subject: NEOPLASIAS / NEUROLOGIA Year: 2020 Document type: Article Affiliation country: Estados Unidos
Fulltext
Add to My VHL
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Cerebellar Neoplasms / Early Intervention, Educational / Medulloblastoma Type of study: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Limits: Child / Child, preschool / Female / Humans / Male Language: En Journal: Neuro Oncol Journal subject: NEOPLASIAS / NEUROLOGIA Year: 2020 Document type: Article Affiliation country: Estados Unidos