NF1-PN Care

Real Patient Living with NF1-PN

Patients with NF1-PN need more options

When I was growing up, I was told ‘there’s nothing we can do’ or to ‘wait and see.’

– Krista, living with NF1-PN

Despite advancements in NF1-PN treatment, unmet needs remain, and patients are in need of more options

Systemic therapy has shown promise in treating neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN), but innovation is still needed1

Currently, adults are limited to off-label therapies and surgery.1-3

There are no approved treatment options for adults with NF1-PN.1

Challenges with options used to manage NF1-PN include1,4:

Not all patients respond

Tolerability challenges

that lead to dose reductions and treatment discontinuation

Limited pediatric-friendly dosing formulations

A lifelong condition like NF1‑PN requires treatment options optimized for continuous use in patients of all ages.1,5

Unresectable tumors, incomplete resections, and tumor regrowth limit the utility of surgical intervention4,6

  • NF1-PN surgery is often restricted by tumor site or infiltration of surrounding nerves and vasculature7
  • Even when surgery is feasible, complete removal of a plexiform neurofibroma (PN) is not possible in many cases8,9
  • Rates of PN regrowth following incomplete resection have been reported to range from 29% to ~70% depending on the extent of resection7,8,*,†
  • 16% (n=5) of patients with NF1-PN who received debulking surgery reported complications2,‡
    • Of these patients, 60% (n=3) were burdened with acute surgical complications, including delayed healing and bleeding, and 40% (n=2) experienced chronic postoperative symptoms, such as functional impairment and nerve damage

~50% of patients

have tumors that cannot be completely resected10

20% of tumors regrow

after a reported complete resection7,†
“We were going to reconstructive surgeons, but I was looking at a long journey to figure out how they were going to debulk the tumors. And then my neurologist said, ‘This is not something you want to start messing with because you’re basically opening Pandora’s box.’”
Real Patient Living with NF1-PN
– Krista, living with NF1-PN

Having more nonsurgical treatment options for patients with NF1-PN is critical.6

NF1-PN can be challenging for young adults

For patients who are transitioning to adulthood, NF1-PN can be particularly burdensome.
LEARN WHY
*
Based on a retrospective analysis of 154 pediatric NF1-PN patients seen at Cincinnati Children’s Hospital Medical Center between 1997 and 2007.8
†
Based on a retrospective review of 121 NF1-PN patients seen at the Children’s Hospital of Philadelphia between 1974 and 1994.7

‡

Based on a cross-sectional study using a one-time survey of NF1-PN patients and caregivers. Sixty-one pediatric patients 8 to 18 years old and their caregivers, and 21 additional caregivers of patients 2 to 7 years old (total of 82 caregivers) participated in the survey.2
  1. Acar S, Armstrong AE, Hirbe AC. Plexiform neurofibroma: shedding light on the investigational agents in clinical trials. Expert Opin Investig Drugs. 2022;31(1):31-40.
  2. Yang X, Yoo HK, Amin S, et al. Clinical and humanistic burden among pediatric patients with neurofibromatosis type 1 and plexiform neurofibroma in the USA. Childs Nerv Syst. 2022;38(8):1513-1522.
  3. Data on file: SpringWorks Therapeutics, Inc.
  4. Metrock LK, Lobbous M, Korf B. An evaluation of selumetinib for the treatment of neurofibromatosis type 1-associated symptomatic, inoperable plexiform neurofibromas. Expert Rev Precis Med Drug Dev. 2021;6(4):239-246.
  5. Children’s Tumor Foundation. Diagnosed with neurofibromatosis type 1 (NF1): a guide for those living with NF1. 2023. Accessed July 19, 2024. https://www.ctf.org/wp-content/uploads/2023/11/CTF-Diagnosed_NF1_Brochure.pdf
  6. Fisher MJ, Blakeley JO, Weiss BD, et al. Management of neurofibromatosis type 1-associated plexiform neurofibromas. Neuro Oncol. 2022;24(11):1827-1844.
  7. Needle MN, Cnaan A, Dattilo J, et al. Prognostic signs in the surgical management of plexiform neurofibroma: the Children’s Hospital of Philadelphia experience, 1974-1994. J Pediatr. 1997;131(5):678-682.
  8. Prada CE, Rangwala FA, Martin LJ, et al. Pediatric plexiform neurofibromas: impact on morbidity and mortality in neurofibromatosis type 1. J Pediatr. 2012;160(3):461-467.
  9. Yang X, Desai K, Agrawal N, et al. Treatment, resource use and costs among pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas. Pediatric Health Med Ther. 2020;11:421-428.
  10. Selumetinib for Treating Symptomatic and Inoperable Plexiform Neurofibromas Associated With Type 1 Neurofibromatosis in Children Aged 3 and Over. National Institute for Health and Care Excellence; May 5, 2022. HST20. Accessed April 11, 2024. https://www.nice.org.uk/guidance/hst20