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Results summary of St. Jude clinical trial: 



Risk-adapted therapy for young children with medulloblastoma (SJYC07)

Why was this study done?

SJYC07 used a new treatment for brain tumors in children under age 5 years. Doctors used a new combination of standard chemotherapy (chemo) drugs. Some children also had radiation therapy.

Radiation therapy is usually given to the brain and spine of children with medulloblastoma. Very young children who get this treatment may have lasting problems with thinking, learning and growing. In this study we delayed or avoided using radiation therapy, when possible.

Chemo is used to try to shrink medulloblastoma in young children. This allows the brain more time to develop before radiation is given. Chemo may also prevent radiation from being needed at all.

The study’s main goals were to:

  • Study how well this treatment works on brain tumors in young children
  • Better identify groups of tumor types
  • Help predict which patients may respond to treatment, and which patients may have a higher risk for relapse (tumor coming back)
  • Learn more about the genetic makeup of brain tumors in young children  

When was this study done?

The study opened in November 2007 and closed in April 2017.

What did the study consist of?

Patients were treated according to their risk group: low risk, intermediate-risk or high risk.

First, surgeons removed as much tumor as possible.

Then all patients had four courses of induction chemo: methotrexate, vincristine, cisplatin and cyclophosphamide. Patients with high-risk disease also received vinblastine.

Next came consolidation therapy based on risk groups:

  • Low-risk patients had 2, 4-week cycles of cyclophosphamide, etoposide and carboplatin.
  • Intermediate-risk patients had focal radiation therapy to the tumor bed
  • High-risk patients had topotecan and cyclophosphamide.

Finally, all patients received cyclophosphamide, topotecan and erlotinib. 

What did we learn from this study?

  • One out of every 3 children on this study was alive 5 years later without evidence of tumor.
  • For children in the low-risk group, the survival rate was 55%; for intermediate-risk, 24.6%; and for high-risk, 16.7%.
  • The risk-adapted approach to treatment did not improve event-free survival for all young children with medulloblastoma.
  • Studying the genetic makeup of these tumors is very important.
  • Some patients with sonic hedgehog (SHH) subgroup tumors had a unique feature in the biochemical markings (called methylation pattern) of their tumors. Those children had improved survival of 51% compared with patients whose tumors did not have the special feature.
  • We found two new subtypes in the SHH subgroup. These were named iSHH-I and iSHH-II. Children with iSHH-II had better event-free survival rates than those with iSHH-I. This outcome occurred without radiation, intraventricular chemo or high-dose chemo. 

What are the next research steps as a result of this study?

By studying the genetic makeup of the tumors, we now better understand medulloblastoma. We will use results of SJYC07 to plan future medulloblastoma trials. What we learned will lead to new ways to classify these tumors and to plan new risk-adapted studies for this disease.

How does this study affect my child?

Every childhood cancer survivor should receive long-term follow-up care. Through the St. Jude After Completion of Care clinic, your child will receive information and guidance for care after treatment. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

For more information

Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

Publication generated from this study:

Risk-adapted therapy for young children with medulloblastoma (SJYC07): therapeutic and molecular outcomes from a multicentre, phase 2 trial. Robinson GW, Rudneva VA, Buchhalter I, Billups CA, Waszak SM, Smith KS, Bowers DC, Bendel A, Fisher PG, Partap S, Crawford JR, Hassall T, Indelicato DJ, Boop F, Klimo P, Sabin ND, Patay Z, Merchant TE, Stewart CF, Orr BA, Korbel JO, Jones DTW, Sharma T, Lichter P, Kool M, Korshunov A, Pfister SM, Gilbertson RJ, Sanders RP, Onar-Thomas A, Ellison DW, Gajjar A, Northcott PA. Lancet Oncol. 2018 Jun;19(6):768-784. doi: 10.1016/S1470-2045(18)30204-3. Epub 2018 May 16.

Clinical Outcomes and Patient-Matched Molecular Composition of Relapsed Medulloblastoma. Kumar R, Smith KS, Deng M, Terhune C, Robinson GW, Orr BA, Liu APY, Lin T, Billups CA, Chintagumpala M, Bowers DC, Hassall TE, Hansford JR, Khuong-Quang DA, Crawford JR, Bendel AE, Gururangan S, Schroeder K, Bouffet E, Bartels U, Fisher MJ, Cohn R, Partap S, Kellie SJ, McCowage G, Paulino AC, Rutkowski S, Fleischhack G, Dhall G, Klesse LJ, Leary S, Nazarian J, Kool M, Wesseling P, Ryzhova M, Zheludkova O, Golanov AV, McLendon RE, Packer RJ, Dunham C, Hukin J, Fouladi M, Faria CC, Pimentel J, Walter AW, Jabado N, Cho YJ, Perreault S, Croul SE, Zapotocky M, Hawkins C, Tabori U, Taylor MD, Pfister SM, Klimo P Jr, Boop FA, Ellison DW, Merchant TE, Onar-Thomas A, Korshunov A, Jones DTW, Gajjar A, Ramaswamy V, Northcott PA. J Clin Oncol. 2021 Mar 1;39(7):807-821. doi: 10.1200/JCO.20.01359. Epub 2021 Jan 27.