학술논문
A multidisciplinary care pathway improves quality of life and reduces pain in patients with fibrous dysplasia/McCune-Albright syndrome: a multicenter prospective observational study.
Document Type
Academic Journal
Author
Meier ME; Center for Bone Quality, Department of Orthopaedic Surgery, Leiden University Medical Center, Leiden, The Netherlands. m.e.meier@lumc.nl.; Hagelstein-Rotman M; Division of Endocrinology, Department of Internal Medicine, Center for Bone Quality, Leiden University Medical Center, Leiden, The Netherlands.; van de Ven AC; Department of Endocrinology, Radboud University Medical Center, Nijmegen, The Netherlands.; Van der Geest ICM; Department of Orthopaedic Surgery, Radboud University Medical Center, Nijmegen, The Netherlands.; Donker O; Center for Bone Quality, Department of Orthopaedic Surgery, Leiden University Medical Center, Leiden, The Netherlands.; Pichardo SEC; Department of Oral and Maxillofacial Surgery, Leiden University Medical Center, Leiden, The Netherlands.; Hissink Muller PCE; Department of Paediatric Rheumatology, Willem Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands.; van der Meeren SW; Department of Ophthalmology, Leiden University Medical Center, Leiden, The Netherlands.; Dorleijn DMJ; Division of Endocrinology, Department of Internal Medicine, Center for Bone Quality, Leiden University Medical Center, Leiden, The Netherlands.; Winter EM; Division of Endocrinology, Department of Internal Medicine, Center for Bone Quality, Leiden University Medical Center, Leiden, The Netherlands.; van de Sande MAJ; Center for Bone Quality, Department of Orthopaedic Surgery, Leiden University Medical Center, Leiden, The Netherlands.; Appelman-Dijkstra NM; Division of Endocrinology, Department of Internal Medicine, Center for Bone Quality, Leiden University Medical Center, Leiden, The Netherlands.
Source
Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE
Subject
Language
English
Abstract
Background: Fibrous dysplasia/McCune-Albright syndrome (FD/MAS) may cause pain, impaired ambulation and decreased quality of life (QoL). International guidelines advocate management of FD/MAS in a tertiary multidisciplinary care pathway, but no longitudinal data are available to support this recommendation. This multicenter prospective observational study aimed to evaluate effects of 1 year of treatment in the FD/MAS care pathway in 2 tertiary clinics on QoL and pain, assessed by change in Short Form 36 and Brief Pain Inventory between baseline and follow-up. Patients completing baseline questionnaires < 1 year after intake were classified as new referrals, others as under chronic care.
Results: 92 patients were included, 61 females (66%). 22 patients (24%) had monostotic disease, 16 (17%) isolated craniofacial FD, 27 (40%) polyostotic FD and 17 (19%) MAS. 26 were new referrals (28%) and 66 chronic patients (72%). Median age at baseline was 47 years (Q1-Q3 36-56). Skeletal burden correlated with baseline Physical Function (rs = - 0.281, p = 0.007). QoL was in all domains lower compared to the general population. New referrals reported clinically important differences (CID) over time in domains Physical Function (mean 67 ± SD24 to 74 ± 21, effect size (ES) 0.31, p = 0.020), Role Physical (39 ± 41 to 53 ± 43, ES 0.35, p = 0.066), Social Functioning (64 ± 24 to 76 ± 23, ES 0.49, p = 0.054), and Health Change (39 ± 19 to 53 ± 24, ES 0.76, p = 0.016), chronic patients in Physical Function (52 ± 46 to 66 ± 43, ES 0.31, p = 0.023) and Emotional Wellbeing (54 ± 27 to 70 ± 15, ES 0.59, p < 0.001). New referrals reported a CID of 1 point in maximum pain, average pain and pain interference, chronic patients reported stable scores. Change in pain interference and Role Physical were correlated (r s = - 0.472, p < 0.001). Patients with limited disease extent improved more than patients with severe disease. Patients receiving FD-related therapy had lower baseline scores than patients not receiving therapy and reported improvements in QoL after 1 year. Yet also patients without FD-related therapy improved in Physical Function.
Conclusions: All FD-subtypes may induce pain and reduced QoL. A multidisciplinary care pathway for FD/MAS may improve pain and QoL, mainly in new referrals without MAS comorbidities with low baseline scores. Therefore, we recommend referral of patients with all subtypes of FD/MAS to specialized academic centers.
(© 2022. The Author(s).)
Results: 92 patients were included, 61 females (66%). 22 patients (24%) had monostotic disease, 16 (17%) isolated craniofacial FD, 27 (40%) polyostotic FD and 17 (19%) MAS. 26 were new referrals (28%) and 66 chronic patients (72%). Median age at baseline was 47 years (Q1-Q3 36-56). Skeletal burden correlated with baseline Physical Function (r
Conclusions: All FD-subtypes may induce pain and reduced QoL. A multidisciplinary care pathway for FD/MAS may improve pain and QoL, mainly in new referrals without MAS comorbidities with low baseline scores. Therefore, we recommend referral of patients with all subtypes of FD/MAS to specialized academic centers.
(© 2022. The Author(s).)