What is your preferred systemic therapy for metastatic malignant peripheral nerve sheath tumor with early relapse after adjuvant AIM?

Given metastatic disease and not a trial candidate, how would you approach this scenario? How does an underlying diagnosis of NF1 affect your decision (if at all)?

1 Answer

Mednet MemberInvited Expert

Medical Oncology · Emory University

Answered on

Persons with MPNST and NF-1 do worse than people without NF-1. I am suspicious that MPNST in persons without NF-1 is actually more than one entity but in NF-1 it is all the same entity.

I have had some success with pazopanib in persons without NF-1. Also I have seen two persons who responded to the combination of cyclophosphamide and sirolimus as described by Schuetze et al. I give sirolimus as 3mg instead of 4mg.

Results of a phase II study of sirolimus and cyclophosphamide in patients with advanced sarcoma.

I have really had no luck with systemic treatment in metastatic MPNST associated with NF-1 despite many efforts. Serial destructive treatments can help, I think; especially with the decreased toxicity of IMRT one can address individual lesions that are producing or threatening symptoms. It seems to me like MPNST in NF-1 has a propensity for brain mets and it would make sense to take a look with MRI; they are easier to treat if caught before they are symptomatic.