Magnitude and relevance of change in health-related quality of life in patients with vascular malformations treated with sirolimus.

Autor:	Harbers VEM; Medical Imaging, Radboud University Medical Center, Nijmegen, Netherlands.; Radboudumc Center of Expertise HECOVAN, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, Netherlands., Bouwman FCM; Radboudumc Center of Expertise HECOVAN, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, Netherlands.; Department of Surgery, Radboud University Medical Center, Nijmegen, Netherlands., van Rijnsoever IMP; Radboudumc Center of Expertise HECOVAN, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, Netherlands.; Department of Pediatrics, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, Netherlands., Verhoeven BH; Radboudumc Center of Expertise HECOVAN, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, Netherlands.; Department of Surgery, Radboud University Medical Center, Nijmegen, Netherlands., van der Vleuten CJM; Radboudumc Center of Expertise HECOVAN, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, Netherlands.; Department of Dermatology, Radboud University Medical Center, Nijmegen, Netherlands.; Members of the Vascular Anomalies Working Group (VASCA WG) of the European Reference Network for Rare Multisystemic Vascular Diseases (VASCERN), Paris, France., Schultze Kool LJ; Medical Imaging, Radboud University Medical Center, Nijmegen, Netherlands.; Radboudumc Center of Expertise HECOVAN, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, Netherlands.; Members of the Vascular Anomalies Working Group (VASCA WG) of the European Reference Network for Rare Multisystemic Vascular Diseases (VASCERN), Paris, France., de Laat PCJ; Department of Pediatric Oncology, WEVAR-Team, Rotterdam Erasmus MC-Sophia, Rotterdam, Netherlands., van der Horst CMAM; Department of Plastic Reconstructive and Hand Surgery, AVA-Team, Amsterdam University Medical Center, Amsterdam, Netherlands., Kievit W; Health Technology Assessment, Department for Health Evidence, Radboud University Medical Center, Nijmegen, Netherlands., Te Loo DMWM; Radboudumc Center of Expertise HECOVAN, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, Netherlands.; Department of Pediatric Hematology, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, Netherlands.
Jazyk:	angličtina
Zdroj:	Frontiers in medicine [Front Med (Lausanne)] 2023 Apr 20; Vol. 10, pp. 1155476. Date of Electronic Publication: 2023 Apr 20 (Print Publication: 2023).
DOI:	10.3389/fmed.2023.1155476
Abstrakt:	Introduction: Vascular malformations are rare congenital anomalies of the vascular system, which can involve the capillaries, veins, arteries, lymphatics, or a combination of vessel types. Patients with vascular malformations experience an impaired health-related quality of life (HRQoL) because of their symptoms (e.g., pain, swelling, and bleeding) and psychosocial distress. Sirolimus is an effective drug used in the medical treatment of these patients; however, relatively little is known about the effect of sirolimus on specific changes in the HRQoL domains and its magnitude. Methods: The magnitude of change (effect size) following intervention is more informative to clinical practitioners than statistically significant but clinically unimportant changes; therefore, this study aimed to examine the magnitude and meaningfulness of change in the HRQoL of children and adults with vascular malformations following sirolimus treatment using low target levels. Results: In total, 50 patients with vascular malformations (19 children, 31 adults) were included in this study. These patients experienced a lower HRQoL than the general population, with the adults reporting a significantly lower score in almost all domains. A 6-month sirolimus treatment improved the HRQoL in 29 patients, including 77.8% of the children (Pediatric Quality of Life Inventory score [PedsQL]) and 57.7% of the adults (Short Form 36 [SF-36]). The effect sizes of sirolimus for each SF-36/PedsQL domain ranged from 0.19 to 1.02. The clinically relevant moderate magnitude of changes was seen in the domains of the children's reports: "Physical functioning" and "Social functioning" and in the domains of the parent reports: "Social functioning," "School functioning," and "Psychosocial." A high-magnitude change was seen in the domains "Emotional functioning" and "Psychosocial" in the children's reports and "Physical functioning" in the parent reports. In addition, the moderate magnitude of changes was also seen in the adults SF-36: in all domains except for "Role limitations-physical problems," "Role limitations-emotional problems," and "General health perception." Conclusion: We believe this is the first study showing the magnitude of change in HRQoL after sirolimus treatment in patients with vascular malformations. Before treatment, these patients experienced an impaired HRQoL compared with the general Dutch population. A 6-month sirolimus treatment with low target levels led to moderate-to-high clinically relevant changes in multiple domains, which significantly improved the HRQoL. Clinical Trial Registration: https://clinicaltrials.gov/ct2/show/NCT03987152?cond=Vascular+Malformations&cntry=NL&city=Nijmegen&draw=2&rank=1, identifier: NCT03987152. Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. (Copyright © 2023 Harbers, Bouwman, van Rijnsoever, Verhoeven, van der Vleuten, Schultze Kool, de Laat, van der Horst, Kievit and te Loo.)
Databáze:	MEDLINE
Externí odkaz:	Zobrazit plný text záznamu