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A controlled multiphase trial of ketotifen to minimize neurofibroma-associated pain and itching
1Neurofibromatosis Institute, La Crescenta, CA 91214.
Background And Design:
Based on potential contributions of mast cells to neurofibroma-associated itching, pain, and tenderness, the mast cell blocker ketotifen fumarate (Zaditen, Sandoz Pharmaceuticals Corp, Hanover, NJ) has been proposed as a treatment for these symptoms. To test the hypothesis that ketotifen decreases neurofibroma-associated itching, pain, and tenderness, data were accumulated from two protocols. The first was an open-label protocol involving 25 patients with relatively severe symptoms (1170 patient-months), and the second was a double-blind protocol involving 27 patients with either relatively mild or severe neurofibroma-associated symptoms (316 patient-months). All subjects received either oral placebo or 2 to 4 mg of ketotifen fumarate per day. Using a scale of 1 to 10, symptoms were measured before, during, and after treatment.
Results:
Itching severity scores (means) were as follows: for all patients receiving ketotifen, 7.8 before, 2.8 during, and 7.2 after treatment; for ketotifen-treated patients in the double-blind protocol, 6.6 before, 3.9 during, and 6.4 after treatment; and for placebo-treated patients, 6.0 before and 6.0 during treatment. Pain and tenderness severity scores (means) were as follows: for all patients treated with ketotifen, 7.6 before, 3.6 during, and 6.6 after treatment; for double-blind ketotifen-treated patients, 6.3 before, 4.6 during, and 6.1 after treatment; and for placebo-treated patients, 7.9 before and 6.7 during treatment.
Conclusions:
Pretreatment, treatment, and posttreatment levels of itching, pain, and tenderness associated with neurofibromas, using both open-label and double-blind protocols, indicate that ketotifen offers a realistic approach to treating these symptoms.
Insights
Ketotifen fumarate significantly reduced itching, pain, and tenderness in neurofibroma patients. This mast cell blocker offers a realistic treatment approach for these debilitating symptoms.
Area of Science:
- Pharmacology
- Dermatology
- Neuro-oncology
Background:
- Neurofibromas are associated with mast cell activity.
- Itching, pain, and tenderness are common neurofibroma symptoms.
- Ketotifen fumarate, a mast cell blocker, is a potential treatment.
Purpose of the Study:
- To evaluate ketotifen fumarate's efficacy in reducing neurofibroma-associated symptoms.
- To test the hypothesis that ketotifen decreases itching, pain, and tenderness.
Main Methods:
- Two protocols were used: an open-label (25 patients) and a double-blind (27 patients).
- Patients received oral placebo or 2-4 mg/day of ketotifen fumarate.
- Symptoms were measured on a 1-10 scale before, during, and after treatment.
Main Results:
- Ketotifen significantly reduced mean itching scores from 7.8 to 2.8.
- Ketotifen significantly reduced mean pain and tenderness scores from 7.6 to 3.6.
- Placebo showed no significant change in symptom severity during treatment.
Conclusions:
- Ketotifen fumarate effectively alleviates itching, pain, and tenderness in neurofibroma patients.
- Both open-label and double-blind data support ketotifen as a viable treatment option.
- Mast cell blockade with ketotifen is a promising therapeutic strategy for neurofibroma symptom management.