Patient acceptable symptom state and minimal clinically important difference for patient-reported outcomes in systemic sclerosis: A secondary analysis of a randomized controlled trial comparing personalized physical therapy to usual care

Camille Daste; François Rannou; Luc Mouthon; Katherine Sanchez; Alexandra Roren; Vincent Tiffreau; Éric Hachulla; Philippe Thoumie; Jean Cabane; Emmanuel Chatelus; Jean Sibilia; Serge Poiraudeau; Christelle Nguyen

doi:10.1016/j.semarthrit.2018.03.013

Patient acceptable symptom state and minimal clinically important difference for patient-reported outcomes in systemic sclerosis: A secondary analysis of a randomized controlled trial comparing personalized physical therapy to usual care

Semin Arthritis Rheum. 2019 Feb;48(4):694-700. doi: 10.1016/j.semarthrit.2018.03.013. Epub 2018 Mar 27.

Authors

Affiliations

¹ AP-HP, Service de Rééducation et de Réadaptation de l'Appareil Locomoteur et des Pathologies du Rachis, Hôpitaux Universitaires Paris Centre-Groupe Hospitalier Cochin, Paris, France; INSERM UMR 1153, Centre de Recherche Épidémiologie et Statistique Paris Sorbonne Cité, ECaMO Team, Paris, France.
² AP-HP, Service de Rééducation et de Réadaptation de l'Appareil Locomoteur et des Pathologies du Rachis, Hôpitaux Universitaires Paris Centre-Groupe Hospitalier Cochin, Paris, France; Sorbonne Paris Cité, Université Paris Descartes, Faculté de Médecine, Paris, France; INSERM UMR 1124, Laboratoire de Pharmacologie, Toxicologie et Signalisation Cellulaire, Faculté des Sciences Fondamentales et Biomédicales, Centre Universitaire des Saints-Pères, Paris, France.
³ Sorbonne Paris Cité, Université Paris Descartes, Faculté de Médecine, Paris, France; AP-HP, Service de Médecine Interne, Centre de Référence des Maladies Rares, Hôpitaux Universitaires Paris Centre-Groupe Hospitalier Cochin, Paris, France.
⁴ AP-HP, Service de Rééducation et de Réadaptation de l'Appareil Locomoteur et des Pathologies du Rachis, Hôpitaux Universitaires Paris Centre-Groupe Hospitalier Cochin, Paris, France.
⁵ Université Lille 2, Lille, France; Service de Médecine Physique et de Réadaptation, CHU de Lille, Lille, France.
⁶ Université Lille 2, Lille, France; Service de Médecine Interne, Centre de Référence des Maladies Auto-immunes et Systémiques Rares, CHU de Lille, Lille, France.
⁷ Sorbonne Paris Cité, Université Paris Pierre et Marie Curie, Faculté de Médecine, Paris, France; AP-HP, Service de Médecine Physique et de Réadaptation, Hôpital Rothschild, Paris, France.
⁸ AP-HP, Service de Médecine Interne, Hôpital Saint-Antoine, Paris, France.
⁹ Hôpitaux Universitaires de Strasbourg, Service de Rhumatologie, Hôpital Hautepierre, Fédération de Médecine Translationnelle de Strasbourg, Strasbourg, France; INSERM UMR 1109, Université de Strasbourg, Strasbourg, France.
¹⁰ AP-HP, Service de Rééducation et de Réadaptation de l'Appareil Locomoteur et des Pathologies du Rachis, Hôpitaux Universitaires Paris Centre-Groupe Hospitalier Cochin, Paris, France; INSERM UMR 1153, Centre de Recherche Épidémiologie et Statistique Paris Sorbonne Cité, ECaMO Team, Paris, France; Sorbonne Paris Cité, Université Paris Descartes, Faculté de Médecine, Paris, France; Institut Fédératif de Recherche sur le Handicap, Paris, France.
¹¹ AP-HP, Service de Rééducation et de Réadaptation de l'Appareil Locomoteur et des Pathologies du Rachis, Hôpitaux Universitaires Paris Centre-Groupe Hospitalier Cochin, Paris, France; Sorbonne Paris Cité, Université Paris Descartes, Faculté de Médecine, Paris, France; INSERM UMR 1124, Laboratoire de Pharmacologie, Toxicologie et Signalisation Cellulaire, Faculté des Sciences Fondamentales et Biomédicales, Centre Universitaire des Saints-Pères, Paris, France. Electronic address: christelle.nguyen2@aphp.fr.

PMID: 29685482
DOI: 10.1016/j.semarthrit.2018.03.013

Abstract

Background: To estimate patient acceptable symptom state (PASS) and minimal clinically important difference (MCID) for patient-reported outcomes in systemic sclerosis (SSc).

Methods: We conducted a secondary analysis of the SCLEREDUC trial, a 12-month randomized controlled trial comparing the efficacy of physical therapy to usual care in 220 SSc patients followed-up from September 2005 to October 2010. Self-rated state and change in patient health at 12 months were assessed by using 2 external anchors extracted from the Medical Outcomes Study 36-Item Short-Form. Patients who self-rated their health as "excellent", "very good" or "good" were the PASS group and those who self-rated their health change as "somewhat better" were the MCID group. Main outcomes were the estimates of PASS by using the 75th percentile method and of MCID by using the mean change in scores method for pain and activity limitation.

Results: PASS (95% confidence interval) and mean (SD) MCID estimates at 12 months were 53.75 (34.00 to 68.00) and -6.74 (32.02) for the joint-pain visual analog scale (range 0-100), 1.41 (1.13 to 1.63) and -0.21 (0.48) for the Health Assessment Questionnaire (HAQ, range 0-3), 1.27 (1.07 to 1.62) and -0.13 (0.45) for the scleroderma HAQ (range 0-3), 26.00 (17.00 to 37.00) and -3.38 (9.87) for the Cochin Hand Function Scale (range 0-90), and 19.40 (17.20 to 21.90) and -5.69 (6.79) for the McMaster-Toronto Arthritis Patient Preference Disability Questionnaire (range 0-30), respectively.

Conclusions: We provide, for the first time, the PASS and MCID estimates for pain and activity limitation in SSc.

Trial registration: ClinicalTrials.gov Identifier: NCT00318188. First Posted: April 26, 2006.

Keywords: Minimal clinically important difference; Patient acceptable symptom state; Patient-reported outcomes; Systemic sclerosis.

Publication types

Comparative Study
Randomized Controlled Trial
Research Support, Non-U.S. Gov't

MeSH terms

Adult
Aged
Female
Humans
Male
Middle Aged
Minimal Clinically Important Difference
Patient Reported Outcome Measures
Patient Satisfaction
Physical Therapy Modalities*
Scleroderma, Systemic / drug therapy
Scleroderma, Systemic / therapy*

Associated data

ClinicalTrials.gov/NCT00318188