Mathieu Puyade1,2, Nancy Maltez3, Pauline Lansiaux4,5, Grégory Pugnet6,7, Pascal Roblot1,8, Ines Colmegna9,10, Marie Hudson10,11, Dominique Farge4,5,10. 1. Centre Hospitalier Universitaire de Poitiers, Service de Médecine Interne, Maladies infectieuses, France. 2. Centre Hospitalier Universitaire de Poitiers, CIC1402, Poitiers, France. 3. Ottawa Hospital, Ottawa, Canada. 4. Unité de Médecine Interne: Maladies Auto-immunes et Pathologie Vasculaire (UF 04), Centre de Référence des Maladies auto-immunes systémiques Rares d'Ile-de-France, AP-HP, Hôpital St-Louis, Saint Louis. 5. Université de Paris, IRSL, Recherche clinique appliquée à l'hématologie, EA 3518, F-75010 Paris. 6. CHU de Toulouse, Hôpital Purpan, Service de Médecine Interne, France. 7. CHU de Toulouse, CIC 1436 module Biothérapie, Toulouse, France. 8. Université de Poitiers, Poitiers, France. 9. Research Institute of the McGill University Health Center, Montreal, Canada. 10. Department of Medicine, McGill University, Montreal, Canada. 11. Jewish General Hospital, Lady Davis Institute, Montreal, Canada.
Abstract
OBJECTIVES: In severe rapidly progressive SSc, autologous haematopoietic stem cell transplantation (AHSCT) allows significant improvements in overall and event-free survival. We undertook this study to identify, appraise and synthesize the evidence on health-related quality of life (HRQoL) before and after AHSCT for SSc. METHODS: We performed a systematic review of the literature, following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines, in PubMed and ScienceDirect from database inception to 1 February 2019. All articles with original HRQoL data were selected. RESULTS: The search identified 1080 articles, of which 8 were selected: 3 unblinded randomized controlled trials [American Scleroderma Stem Cell versus Immune Suppression Trial (ASSIST), Autologous Stem Cell Transplantation International Scleroderma, Scleroderma: Cyclophosphamide or Transplantation), 3 uncontrolled phase I or II trials and 2 cohort studies. HRQoL data from 289 SSc patients treated with AHSCT and 125 treated with intravenous CYC as a comparator with median 1.25-4.5 years follow-up were included. HRQoL was evaluated with the HAQ Disability Index (HAQ-DI; 275 patients), the 36-item Short Form Health Survey (SF-36; 249 patients) and the European Quality of Life 5-Dimensions questionnaire (EQ-5D; 138 patients). The quality of the studies was moderate to low. AHSCT was associated with significant improvement in the HAQ-DI (P = 0.02-<0.001), SF-36 Physical Component Summary score (P = 0.02-<0.0001) and EQ-5D index-based utility score (P < 0.001). The SF-36 Mental Component Summary score improved in the ASSIST (n = 19) and one small retrospective cohort (n = 30 patients, P = 0.005) but did not improve significantly in 2 randomized controlled trials (n = 200 patients, P = 0.1-0.91). CONCLUSION: AHSCT in severe SSc patients is associated with significant and durable improvement in physical HRQoL.
OBJECTIVES: In severe rapidly progressive SSc, autologous haematopoietic stem cell transplantation (AHSCT) allows significant improvements in overall and event-free survival. We undertook this study to identify, appraise and synthesize the evidence on health-related quality of life (HRQoL) before and after AHSCT for SSc. METHODS: We performed a systematic review of the literature, following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines, in PubMed and ScienceDirect from database inception to 1 February 2019. All articles with original HRQoL data were selected. RESULTS: The search identified 1080 articles, of which 8 were selected: 3 unblinded randomized controlled trials [American Scleroderma Stem Cell versus Immune Suppression Trial (ASSIST), Autologous Stem Cell Transplantation International Scleroderma, Scleroderma: Cyclophosphamide or Transplantation), 3 uncontrolled phase I or II trials and 2 cohort studies. HRQoL data from 289 SSc patients treated with AHSCT and 125 treated with intravenous CYC as a comparator with median 1.25-4.5 years follow-up were included. HRQoL was evaluated with the HAQ Disability Index (HAQ-DI; 275 patients), the 36-item Short Form Health Survey (SF-36; 249 patients) and the European Quality of Life 5-Dimensions questionnaire (EQ-5D; 138 patients). The quality of the studies was moderate to low. AHSCT was associated with significant improvement in the HAQ-DI (P = 0.02-<0.001), SF-36 Physical Component Summary score (P = 0.02-<0.0001) and EQ-5D index-based utility score (P < 0.001). The SF-36 Mental Component Summary score improved in the ASSIST (n = 19) and one small retrospective cohort (n = 30 patients, P = 0.005) but did not improve significantly in 2 randomized controlled trials (n = 200 patients, P = 0.1-0.91). CONCLUSION: AHSCT in severe SSc patients is associated with significant and durable improvement in physical HRQoL.
Authors: Sebastian Bruera; Harish Sidanmat; Donald A Molony; Maureen D Mayes; Maria E Suarez-Almazor; Kate Krause; Maria Angeles Lopez-Olivo Journal: Cochrane Database Syst Rev Date: 2022-07-29
Authors: Marianna Y Kawashima-Vasconcelos; Maynara Santana-Gonçalves; Djúlio C Zanin-Silva; Kelen C R Malmegrim; Maria Carolina Oliveira Journal: Front Immunol Date: 2022-08-11 Impact factor: 8.786