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Desmoplastic nodular medulloblastoma in young children: a management dilemma
Mohamed S AbdelBaki1, Daniel R Boué2, Jonathan L Finlay1
1Division of Hematology, Oncology and Bone Marrow Transplant, Nationwide Children's Hospital and The Ohio State University, Columbus, Ohio.
Background:
Children with desmoplastic nodular medulloblastoma (DNMB) have excellent survival, leading multiple groups globally to attempt reduction of treatment-related morbidity. In 2013, the Children's Oncology Group began a clinical trial (ACNS1221) eliminating both radiation therapy (RT) and intraventricular methotrexate for children under 3 years of age with localized DNMB, aiming to build upon the excellent outcomes of the German HIT trials. ACNS1221 has recently closed due to increased incidence of recurrences noted at the 2-year interim analysis, raising important questions regarding optimal therapy for DNMB.
Methods:
A review of major clinical trials that included children with DNMB was performed through July 2017.
Results:
One hundred and eighty-eight DNMB patients enrolled on 11 prospective clinical trials were identified. The use of marrow-ablative chemotherapy and autologous hematopoietic cell rescue (AuHCR) or treatment with intraventricular methotrexate has been associated with excellent outcomes. RT was usually required for patients with evidence of disease at the end of therapy.
Conclusions:
The minimal intensity and duration of chemotherapy required to maximally cure children with DNMB without need of RT remains unknown. Further trials are required to better identify a subset of DNMB patients who can be cured without marrow-ablative chemotherapy or intraventricular methotrexate.
Insights
Treatment for desmoplastic nodular medulloblastoma (DNMB) in young children is being re-evaluated. Eliminating radiation therapy and intraventricular methotrexate led to increased recurrences, prompting further research into optimal chemotherapy regimens.
Area of Science:
- Pediatric Oncology
- Neuro-Oncology
- Clinical Trials
Background:
- Desmoplastic nodular medulloblastoma (DNMB) in children has a high survival rate.
- Treatment-related morbidity reduction is a key goal for DNMB management.
- A recent clinical trial (ACNS1221) for young children (<3 years) with localized DNMB omitted radiation therapy (RT) and intraventricular methotrexate.
Purpose of the Study:
- To evaluate the outcomes of DNMB treatment strategies.
- To determine the optimal intensity and duration of chemotherapy for DNMB.
- To identify DNMB patient subsets curable without intensive treatments like RT or intraventricular methotrexate.
Main Methods:
- A comprehensive review of major clinical trials involving DNMB patients was conducted up to July 2017.
- Data from 188 DNMB patients across 11 prospective clinical trials were analyzed.
Main Results:
- Marrow-ablative chemotherapy with autologous hematopoietic cell rescue (AuHCR) and intraventricular methotrexate are associated with excellent outcomes in DNMB.
- Radiation therapy was generally necessary for patients with residual disease post-treatment.
- The ACNS1221 trial was closed due to a higher incidence of recurrence.
Conclusions:
- The optimal chemotherapy regimen for curing DNMB without causing significant long-term side effects remains undetermined.
- Further clinical trials are essential to identify DNMB patients who can be effectively treated with less intensive chemotherapy regimens.
- Minimizing treatment intensity while maintaining high cure rates for DNMB is an ongoing challenge.
