Current Utilization of Mast Cell Stabilizers for Preemptive Treatment of NF1 Neurofibromas

Abstract

Received: September 9th, 2015 Accepted: September 19th, 2015 The morbidity and mortality of Neurofibromatosis type I (NF1) are both largely re- Published: September 21st, 2015 lated to the person’s neurofibroma burden. That burden can presently be minimized by mast cell stabilizers, with ketotifen as the one most frequently considered for NF1 patients in the pub- lished literature. Here, I review pertinent clinical and research publications to 1) document the Citation rationale for using mast cell blockers in NF1, 2) consider the NF1 clinical impact of mast cell Riccardi VM. Current utilization of blockers, 3) document the relative safety and very modest expense of (at least some) mast cell mast cell stabilizers for preemptive treatment of NF1 neurofibromas. blockers, and 4) suggest that the data are sufficiently robust to support the regular, if not routine Neuro Open J. 2015; 2(2): 67-73. use of mast cell blockers to treat NF1, particularly in children, while the NF1 neurofibroma burden is the least it will be. The rationale for these salutary results have been established by histopathology, Nf1+/- mouse models, a series of open-label and double-blind protocols, , compelling case reports and a series of patients who have afforded their own self-determined mast cell stabilizer treatment (most often ketotifen). In addition, in the intervening 20-plus years since the first formal protocol publications, the positive treatment results have never been refuted or contradicted. The results of the mast-cell-stabilizing treatment are designed to keep the NF1 neurofibroma burden and its consequences at their minimums, in effect preempting NF1 neurofibroma initiation and progression as much as possible.