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Observational Study
. 2022 Dec 17;17(1):439.
doi: 10.1186/s13023-022-02588-z.

A multidisciplinary care pathway improves quality of life and reduces pain in patients with fibrous dysplasia/McCune-Albright syndrome: a multicenter prospective observational study

Maartje E Meier  1 , Marlous Hagelstein-Rotman  2 , Annenienke C van de Ven  3 , Ingrid C M Van der Geest  4 , Olav Donker  5 , Sarina E C Pichardo  6 , Petra C E Hissink Muller  7 , Stijn W van der Meeren  8 , Desirée M J Dorleijn  2 , Elizabeth M Winter  2 , Michiel A J van de Sande  5 , Natasha M Appelman-Dijkstra  2
Affiliations
Observational Study

A multidisciplinary care pathway improves quality of life and reduces pain in patients with fibrous dysplasia/McCune-Albright syndrome: a multicenter prospective observational study

Maartje E Meier et al. Orphanet J Rare Dis. .

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 (rs = - 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.

Keywords: Care pathway; Fibrous dysplasia; McCune-Albright syndrome; Multidisciplinary care; Pain; Patient reported outcome measure; Quality of life; Rare bone disease; Tertiary referral hospital.

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Conflict of interest statement

The authors declare that they have no competing interests.

Figures

Fig. 1
Fig. 1
SF-36 domains at baseline and follow-up in new and chronic patients
Fig. 2
Fig. 2
Pain scores at baseline and follow-up in new and chronic patients with moderate to severe pain
Fig. 3
Fig. 3
Pain scores at baseline and follow-up in new and chronic patients with pain score > 0
See this image and copyright information in PMC

References

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