Nonsurgical treatments may be effective option for cutaneous neurofibromas

Key takeaways:

• Small cNFs were successfully reduced with four different nonsurgical treatment modalities.
• Deoxycholate and alexandrite laser were both effective and the least painful options studied.

Minimally invasive, nonsurgical local treatments could be a possible option for small cutaneous neurofibromas instead of surgical removal, according to a study.

“People with neurofibromatosis type 1 (NF1) develop hundreds to thousands of disfiguring cutaneous neurofibromas (cNFs), with a resultant negative impact on quality of life,” Patricia M. Richey, MD, researcher at Wellman Center for Photomedicine at Massachusetts General Hospital, director of Mohs surgery at Boston University, visiting professor at Harvard Medical School and the study’s first author, told Healio.

“Surgical removal of cNFs under anesthesia, which is most often not covered by insurance, is the only standard therapy and results in scarring,” Richey continued. “Our goal was to develop effective, minimally invasive, safe, rapid, tolerable treatments of cNFs that could potentially prevent tumor progression.”

This prospective, randomized, direct comparison trial included 19 patients who each had a minimum of six cNFs between 2 mm and 4 mm in diameter on the trunk, arms or legs.

Tumors were randomly assigned to be a control or to receive one of four treatment options: deoxycholate solution 10 mg/mL (Kybella, Allergan/AbbVie) injected via a 30-guage needle at approximately the same value as tumor volume; a 755 nm alexandrite laser (Gentlemax Pro, Candela) delivered without dynamic cryogen cooling at 8 mm spot size; a 980 mm diode laser (LightForce Therapy Lasers) delivered via 8 mm passively cooled sapphire window; and radiofrequency needle coagulation, which included an injection with a 27-gauge insulated needle in the mid-dermis that created a pathway for an electrode.

Subjects with at least 24 eligible cNFs received treatment with all four modalities.

A total of 309 cNFs were treated with all but one participant being treated with all four modalities.

Statistically significant reductions in the height and volume of the tumors were observed in all four treatment modalities at 3 months post-treatment.

Mean percentage change in tumor height was –30 with deoxycholate, –26.4 with alexandrite laser, –12.9 with 980 nm laser and –21.8 with radiofrequency. Mean volume change at 6 months was –29.4 with deoxycholate, –33.4 with alexandrite laser, –24.9 with 980 nm laser and –23.3 with radiofrequency.

The most common adverse events were local erythema and edema immediately after treatment. Hypopigmentation was still present in 26% of patients at the alexandrite laser sites. Deoxycholate injection and alexandrite laser were the least painful, according to intraoperative pain scores, with 980 nm laser being the most painful.

“While device-based treatments have been used ad-hoc for treating NF1-associated cNFs, this is the first study to prospectively compare minimally invasive treatment modalities with the intent of developing treatment(s) for early-stage small cNFs. Prior to this, no treatments had been specifically designed for NF1-associated cNFs,” Richey told Healio. “With this study, we showed that partial reduction to complete clearance of small cNFs after one minimally invasive, non-scarring and rapid local treatment is possible.”

More trials are planned, focusing on dose and delivery optimization, wide treatment ranges and the inclusion of younger patients, she added.