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Canna~Fangled Abstracts

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

By December 3, 2024December 4th, 2024No Comments

. 2024 Dec 3;14(1):30105.

doi: 10.1038/s41598-024-79239-9.

Thibaud Lebret 1, Séverine Hatton 1, Jacques Callebert 2 3, Valérie Cormier-Daire 4 5, Céline Greco 6 7
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Abstract

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.

Keywords: Cannabinoids, Chronic pain, Nail-patella syndrome, Noradrenaline, Pain management, Serotonin

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Conflict of interest statement

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.

References

    1. Geerts-Crabbe, L. et al. Difficulties adapting to nail-Patella syndrome: a qualitative study of patients’ perspectives. J. Genet. Couns. 28, 1011–1020. https://doi.org/10.1002/jgc4.1153 (2019). – DOI – PubMed
    1. Bongers, E. M., Gubler, M. C. & Knoers, N. V. Nail-patella syndrome. Overview on clinical and molecular findings. Pediatr. Nephrol. 17, 703–712. https://doi.org/10.1007/s00467-002-0911-5 (2002). – DOI – PubMed
    1. Sweeney, E. et al. in GeneReviews((R)) (eds M. P. Adam (1993).
    1. Witzgall, R. Nail-patella syndrome. Pflugers Arch. 469, 927–936. https://doi.org/10.1007/s00424-017-2013-z (2017). – DOI – PubMed
    1. Lopez-Arvizu, C. et al. Increased symptoms of attention deficit hyperactivity disorder and major depressive disorder symptoms in nail-patella syndrome: potential association with LMX1B loss-of-function. Am. J. Med. Genet. B Neuropsychiatr Genet. 156B, 59–66. https://doi.org/10.1002/ajmg.b.31138 (2011). – DOI – PubMed

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