Improved health-related quality of life in patients treated with topical sirolimus for facial angiofibroma associated with tuberous sclerosis complex
| Autor: | Yuki Ohno, Takashi Hatano, Mayumi Tamari, Shin Egawa, Yu Imai, Jun Moritake, Katsuhisa Endo |
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| Rok vydání: | 2020 |
| Předmět: |
Adult
medicine.medical_specialty Adolescent Facial angiofibromas Health-related quality of life lcsh:Medicine Angiofibroma 030207 dermatology & venereal diseases 03 medical and health sciences Tuberous sclerosis Young Adult 0302 clinical medicine Quality of life Tuberous Sclerosis Internal medicine medicine Humans Pharmacology (medical) In patient 030212 general & internal medicine Adverse effect Genetics (clinical) Topical sirolimus Health related quality of life Sirolimus business.industry Research lcsh:R General Medicine Middle Aged medicine.disease Tuberous sclerosis complex Facial angiofibroma Quality of Life Topical Sirolimus Mental health Facial Neoplasms business medicine.drug |
| Zdroj: | Orphanet Journal of Rare Diseases Orphanet Journal of Rare Diseases, Vol 15, Iss 1, Pp 1-7 (2020) |
| Popis: | Background Tuberous sclerosis complex (TSC) is a rare autosomal dominant disorder forming hamartomas throughout the body. Facial angiofibromas (FAs) occur in 75% of TSC patients, which are often enlarged, impairing the appearance of the face, and reducing the patient’s quality of life (QOL). The aim of this study was to characterize the impact of topical sirolimus treatment on the health-related QOL in patients with FA associated with TSC. Methods We investigated a total of 33 patients who received sirolimus gel treatment for FA associated with TSC and assessed the changes in the health-related QOL using the Medical Outcomes Study 36-Item Short Form (SF-36) Health Survey. SF-36 surveys were performed before and after 3 months of treatment. The conditions of the patients after using the sirolimus gel were categorized into the following three categories: “improved,” “unchanged,” and “aggravated.” Adverse events were investigated using the CTCAE v5.0-JCOG. Results The median age of the patients was 25 (range 14–55) years. After 3 months of sirolimus gel treatment, three scale scores of the SF-36, vitality (VT), social function (SF), and mental health (MH), were significantly improved compared to before the treatment. The VT and SF in patients who had improved FA were significantly better than those in the other patients. There were no significant differences in any scale scores between patients with and without adverse events at 3 months after the initiation of sirolimus gel treatment. Conclusions This is the first report regarding improved health-related quality of life in patients treated with sirolimus gel for FA associated with TSC by using the SF-36. The three scale scores associated with mental health were significantly improved compared to before the treatment. The health-related QOL in patients receiving sirolimus gel treatment is more strongly affected by the treatment efficacy than adverse events. Mini-abstract Sirolimus gel treatment improves the health-related QOL in patients with FA associated with TSC. |
| Databáze: | OpenAIRE |
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