Journal article
SARC006: Phase II Trial of Chemotherapy in Sporadic and Neurofibromatosis Type 1 Associated Chemotherapy-Naive Malignant Peripheral Nerve Sheath Tumors
Sarcoma, Vol.2017, pp.1-8
2017
DOI: 10.1155/2017/8685638
PMCID: PMC5613633
PMID: 29138631
Abstract
Background
. Worse chemotherapy response for neurofibromatosis type 1- (NF1-) associated compared to sporadic malignant peripheral nerve sheath tumors (MPNST) has been reported.
Methods
. We evaluated the objective response (OR) rate of patients with AJCC Stage III/IV chemotherapy-naive NF1 MPNST versus sporadic MPNST after 4 cycles of neoadjuvant chemotherapy, 2 cycles of ifosfamide/doxorubicin, and 2 cycles of ifosfamide/etoposide. A Simon optimal two-stage design was used (target response rate 40%).
Results
. 34 NF1 (median age 33 years) and 14 sporadic (median age 40 years) MPNST patients enrolled. Five of 28 (17.9%) evaluable NF1 MPNST patients had a partial response (PR), as did 4 of 9 (44.4%) patients with sporadic MPNST. Stable disease (SD) was achieved in 22 NF1 and 4 sporadic MPNST patients. In both strata, results in the initial stages met criteria for expansion of enrollment. Only 1 additional PR was observed in the expanded NF1 stratum. Enrollment was slower than expected and the trial closed before full accrual.
Conclusions
. This trial was not powered to detect differences in response rates between NF1 and sporadic MPNST. While the OR rate was lower in NF1 compared to sporadic MPNST, qualitative responses were similar, and disease stabilization was achieved in most patients.
Details
- Title: Subtitle
- SARC006: Phase II Trial of Chemotherapy in Sporadic and Neurofibromatosis Type 1 Associated Chemotherapy-Naive Malignant Peripheral Nerve Sheath Tumors
- Creators
- Christine S. Higham - National Institutes of HealthSeth M. Steinberg - National Institutes of HealthEva Dombi - National Institutes of HealthArie Perry - University of California, San FranciscoLee J. Helman - National Institutes of HealthScott M. Schuetze - University of MichiganJoseph A. Ludwig - The University of Texas MD Anderson Cancer CenterArthur Staddon - University of PennsylvaniaMohammed M. Milhem - University of IowaDaniel Rushing - Indiana UniversityRobin L. Jones - University of WashingtonMichael Livingston - Levine Cancer InstituteStewart Goldman - Lurie Children's HospitalChristopher Moertel - University of MinnesotaLars Wagner - Cincinnati Children's Hospital Medical CenterDavid Janhofer - National Institutes of HealthChristina M. Annunziata - National Institutes of HealthDenise Reinke - Sons of the American RevolutionLauren Long - National Institutes of HealthDavid Viskochil - University of UtahLarry Baker - University of MichiganBrigitte C. Widemann - National Institutes of Health
- Resource Type
- Journal article
- Publication Details
- Sarcoma, Vol.2017, pp.1-8
- DOI
- 10.1155/2017/8685638
- PMID
- 29138631
- PMCID
- PMC5613633
- NLM abbreviation
- Sarcoma
- ISSN
- 1357-714X
- eISSN
- 1369-1643
- Grant note
- DOI: 10.13039/100003192, name: Neurofibromatosis, award: W81XWH-06-1-0434
- Language
- English
- Date published
- 2017
- Academic Unit
- Hematology, Oncology, and Blood & Marrow Transplantation; Internal Medicine
- Record Identifier
- 9984359575702771
Metrics
19 Record Views