Related Experiment Video
Updated: Jul 8, 2026
A Protocol for Rapid Post-mortem Cell Culture of Diffuse Intrinsic Pontine Glioma (DIPG)
Published on: March 7, 2017
Diffuse pontine gliomas in children: changing strategies, changing results? A mono-institutional 20-year experience
Maura Massimino1, Filippo Spreafico, Veronica Biassoni
1Pediatric Oncology Unit, Fondazione IRRCCS Istituto Nazionale Tumori, Via Venezian 1, 20133 Milan, Italy. maura.massimino@istitutotumori.mi.it
Abstract:
Patients with diffuse pontine gliomas have a median survival of less than one year and represent a challenge for pediatric oncologists, prompting them to attempt experimental therapies. From 1987 to 2005, 62 children with diffuse pontine glioma, not amenable to curative surgery, were treated according to four successive pilot protocols: (1) concomitant chemo-radiotherapy (etoposide, cytarabine, ifosfamide, cisplatin, and dactinomycin); (2) intensive high-dose courses chemotherapy (cisplatin/etoposide, cyclophosphamide/vincristine/methotrexate) and a subsequent course of myeloablative thiotepa followed by radiation and maintenance chemotherapy; (3) cisplatin/etoposide followed by isotretinoin before, during and after focal irradiation; and (4) iv vinorelbine before, during, and after irradiation. Considering all patients, 77% experienced a transient response to treatment, always detectable after radiotherapy. The progression-free survival (PFS) rate was 25 +/- 6% at one year, median PFS was seven months; overall survival (OS) was 45 +/- 6%, median OS was eleven months: no statistical differences in the four studies in terms of outcome were detected. Despite improved diagnostic, therapeutic, and supportive tools in pediatric neuro-oncology, little has been achieved for patients with diffuse pontine tumors.
Insights
Pediatric diffuse pontine gliomas remain challenging, with experimental therapies showing limited impact. Despite various treatments, survival rates for these pediatric brain tumors have not significantly improved.
Area of Science:
- Pediatric Neuro-Oncology
- Brain Tumor Research
- Clinical Trial Design
Background:
- Diffuse pontine gliomas (DPGs) present a significant challenge in pediatric oncology.
- Median survival for DPG patients is less than one year, necessitating novel therapeutic approaches.
- Surgical resection is often not feasible for DPGs, limiting curative options.
Purpose of the Study:
- To evaluate the efficacy of four distinct pilot treatment protocols for pediatric diffuse pontine gliomas.
- To assess treatment response, progression-free survival (PFS), and overall survival (OS) in children with DPGs.
- To identify potential improvements in managing this aggressive pediatric brain tumor.
Main Methods:
- A cohort of 62 children with unresectable DPGs were treated across four sequential pilot protocols from 1987 to 2005.
- Protocols included combinations of chemotherapy (etoposide, cytarabine, ifosfamide, cisplatin, dactinomycin, cyclophosphamide, vincristine, methotrexate, thiotepa, vinorelbine) and radiotherapy.
- Specific protocols involved concomitant chemo-radiotherapy, intensive chemotherapy with myeloablation, and targeted agents like isotretinoin.
Main Results:
- A transient treatment response was observed in 77% of patients, typically after radiotherapy.
- One-year progression-free survival (PFS) was 25 +/- 6%, with a median PFS of seven months.
- One-year overall survival (OS) was 45 +/- 6%, with a median OS of eleven months; no significant differences were found between the four protocols.
Conclusions:
- Despite advancements in pediatric neuro-oncology, diffuse pontine tumors remain difficult to treat effectively.
- The evaluated experimental therapies did not yield statistically significant improvements in PFS or OS for DPG patients.
- Further research is critical to develop more effective treatment strategies for this challenging pediatric brain tumor.
