Dataset Information

Pilot study of systemic and intrathecal mafosfamide followed by conformal radiation for infants with intracranial central nervous system tumors: a pediatric brain tumor consortium study (PBTC-001).

ABSTRACT: A pilot study to investigate the feasibility of the addition of intrathecal (IT) mafosfamide to a regimen of concomitant multi-agent systemic chemotherapy followed by conformal radiation therapy (RT) for children <3 years with newly diagnosed embryonal CNS tumors was performed. Ninety-three newly diagnosed infants and children (<3 years) with embryonal CNS tumors were enrolled. Twenty weeks of systemic multi-agent chemotherapy commenced within 35 days of surgery. Patients without CSF flow obstruction (n = 71) received IT mafosfamide (14 mg) with chemotherapy. Localized (M(0)) patients with SD or better subsequently received RT followed by 20 additional weeks of chemotherapy. Second look surgery was encouraged prior to RT if there was an incomplete surgical resection at diagnosis. 71 evaluable patients with normal CSF flow received IT Mafosfamide with systemic chemotherapy; patients with M + disease were removed from protocol therapy at 20 weeks and those with PD at the time of progression. One and 5-year progression free survival (PFS) and overall survival (OS) for the cohort of 71 evaluable patients were 52 ± 6.5 % and 33 ± 13 %, and 67 ± 6.2 % and 51 ± 11 %, respectively. The 1-year Progression Free Survival (PFS) for M0 patients with medulloblastoma (MB, n = 20), supratentorial primitive neuroectodermal tumor (PNET, n = 9), and atypical teratoid rhabdoid tumor (ATRT, n = 12) was 80 ± 7 %, 67 ± 15 % and 27 ± 13 % and 5-year PFS was 65 ± 19 %, 37 ± 29 %, and 0 ± 0 %, respectively. The addition of IT mafosfamide to systemic chemotherapy in infants with embryonal CNS tumors was feasible. The PFS for M0 patients appears comparable to or better than most prior historical comparisons and was excellent for those receiving conformal radiotherapy.

SUBMITTER: Blaney SM

PROVIDER: S-EPMC3529096 | biostudies-literature | 2012 Sep

REPOSITORIES: biostudies-literature

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Pilot study of systemic and intrathecal mafosfamide followed by conformal radiation for infants with intracranial central nervous system tumors: a pediatric brain tumor consortium study (PBTC-001).

Blaney Susan M SM Kocak Mehmet M Gajjar Amar A Chintagumpala Murali M Merchant Thomas T Kieran Mark M Pollack Ian F IF Gururangan Sri S Geyer Russ R Phillips Peter P McLendon Roger E RE Packer Roger R Goldman Stewart S Banerjee Anu A Heideman Richard R Boyett James M JM Kun Larry L

Journal of neuro-oncology 20120713 3

A pilot study to investigate the feasibility of the addition of intrathecal (IT) mafosfamide to a regimen of concomitant multi-agent systemic chemotherapy followed by conformal radiation therapy (RT) for children <3 years with newly diagnosed embryonal CNS tumors was performed. Ninety-three newly diagnosed infants and children (<3 years) with embryonal CNS tumors were enrolled. Twenty weeks of systemic multi-agent chemotherapy commenced within 35 days of surgery. Patients without CSF flow obstru ...[more]

PMID: 22790443

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Project description:Abstract BACKGROUND Optune-delivered TTFields is a promising anti-mitotic FDA-approved cancer therapy in adult patients with high-grade gliomas (HGG), although effectiveness is not yet proven in pediatric patients. A multicenter trial [NCT03033992] evaluating the feasibility and device-related toxicity for children with recurrent or progressive supratentorial high-grade glioma (HGG) and ependymoma is being conducted. This presentation reports the interim health-related quality of life (HRQOL) and device-use experiences reported by patients and parents. METHODS Participating patients (aged 5–21 years) and one of their parents completed the following measures at baseline and prior to each intervention cycle: PROMIS Fatigue, Anger, Anxiety, Depressive Symptoms, Mobility, Upper Extremity Function, and Peer Relationships and Neuro-QOL Stigma. Participants completed an exit survey when they were off the study regarding their experiences wearing the device. RESULTS This planned interim analysis included 11 patients (7 males/4 females) with supratentorial tumors: ten HGG and one ependymoma. Median age was 14.2 (6.4–21.3) years. Patients reported worse HRQOL than the norms on all domains. Of these 11 patients, 6 completed the exit survey. No significant changes (p< 0.05) between the baseline and the final assessment were found except for Stigma. Patients perceived less stigma at baseline than at the final assessment (t=2.82, p=0.0370). Of these 6 parents who completed the Exit Survey: most considered wearing the device to be easy (n=5).Patient reminders were unnecessary (n=5), patients rarely/didn’t complain (n=4) or refuse (n=5) to wear the device, there was rare/no (n=4) difficulty with daily activities because of the device, and patients were not (n=4) or sometimes (n=2) embarrassed to wear the device. CONCLUSION Preliminary results indicate Optune-delivered TTFields therapy is feasible and accepted by children and parent with no evidence of negatively impacting patients’ QOL. Accrual has been expanded beyond the interim analysis.

Dataset Information

Pilot study of systemic and intrathecal mafosfamide followed by conformal radiation for infants with intracranial central nervous system tumors: a pediatric brain tumor consortium study (PBTC-001).

Publications

Pilot study of systemic and intrathecal mafosfamide followed by conformal radiation for infants with intracranial central nervous system tumors: a pediatric brain tumor consortium study (PBTC-001).

Similar Datasets

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