Evidence for therapeutic use of cannabidiol for nail-patella syndrome-induced pain in a real-world pilot study.

Autor: Lebret T; Department of Pain and Palliative Care, Hôpital Necker-Enfants Malades, AP-HP, 149 rue de Sèvres, Paris, France., Hatton S; Department of Pain and Palliative Care, Hôpital Necker-Enfants Malades, AP-HP, 149 rue de Sèvres, Paris, France., Callebert J; Laboratoire de Biochimie et Biologie Moléculaire, Hôpital Lariboisière, AP-HP, Paris, France.; Université Paris Cité, INSERM UMR-S 1144, Optimisation Thérapeutique en Neuropsychopharmacologie, Paris, France., Cormier-Daire V; Service de médecine génomique des maladies rares, Centre de référence des maladies osseuses constitutionnelles, Hôpital Necker-Enfants Malades, AP-HP, Paris, France.; Université Paris Cité, INSERM UMR 1163, Institut Imagine, Paris, France., Greco C; Department of Pain and Palliative Care, Hôpital Necker-Enfants Malades, AP-HP, 149 rue de Sèvres, Paris, France. celine.greco@inserm.fr.; Université Paris Cité, INSERM UMR 1163, Institut Imagine, Paris, France. celine.greco@inserm.fr.
Jazyk: angličtina
Zdroj: Scientific reports [Sci Rep] 2024 Dec 03; Vol. 14 (1), pp. 30105. Date of Electronic Publication: 2024 Dec 03.
DOI: 10.1038/s41598-024-79239-9
Abstrakt: Nail-patella syndrome (NPS) is a rare genetic disease characterized by dysplastic nails, patella abnormalities, skeletal malformation, and chronic pain. Although chronic pain in NPS is mainly due to bone and musculoskeletal symptoms, it can also result from neurological dysfunction. Conventional analgesics are often insufficient to relieve NPS-associated chronic pain. Cannabinoids, which act on the serotonergic and/or noradrenergic pain systems, may therefore represent valuable non-psychoactive alternatives for managing pain in these patients. The effectiveness and safety of synthetic cannabidiol (CBD) for the management of NPS-associated pain was assessed using real-world data from a pilot cohort of patients with NPS who received a 3-month treatment with oral CBD. The treatment (median dose of 900 mg/day) was associated with a significant reduction in pain intensity (mean score of 7.04 ± 0.24 at initiation versus 4.04 ± 0.38 at 3 months, N = 28, p < 0.0001), which correlated with changes in the peripheral concentration of noradrenaline (r = 0.705, 95% CI [0.44-0.86], p < 0.0001). Health-related quality of life and other NPS-associated symptoms also improved in most patients. CBD treatment was well tolerated and no elevations in liver enzyme levels were reported. Synthetic CBD therefore appears to be a safe and effective treatment option for managing NPS-associated chronic pain.
Competing Interests: Declarations. Competing interests: The authors declare no competing interests. Informed consent: All the patients enrolled in this prospective study, or their legal guardian for patients aged ≤ 18 years, provided written informed consent for the publication of their data included in this article. Institutional review board Statement: The institutional review board of the reference center for constitutional bone diseases, the MOC-Necker-Hospital (Paris, France), approved the study. In line with the French legislation on studies conducted in routine clinical practice, the study protocol was registered by the Assistance Publique–Hôpitaux de Paris the 16/05/2022 under the following reference: MR004-2022-0516123418.
(© 2024. The Author(s).)
Databáze: MEDLINE
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