Association of Autologous Hematopoietic Stem Cell Transplantation in Systemic Sclerosis With Marked Improvement in Health-Related Quality of Life.


Journal

Arthritis & rheumatology (Hoboken, N.J.)
ISSN: 2326-5205
Titre abrégé: Arthritis Rheumatol
Pays: United States
ID NLM: 101623795

Informations de publication

Date de publication:
02 2021
Historique:
received: 25 02 2020
revised: 18 08 2020
accepted: 03 09 2020
pubmed: 11 9 2020
medline: 2 3 2021
entrez: 10 9 2020
Statut: ppublish

Résumé

To quantify the magnitude, domains, and duration of change in health-related quality of life (HRQoL) in patients with systemic sclerosis (SSc) who underwent autologous hematopoietic stem cell transplantation (HSCT) as compared to SSc patients with similar characteristics who did not undergo autologous HSCT. The study was designed as a retrospective study comparing SSc patients who underwent autologous HSCT and SSc patients who met the criteria for transplantation but were treated with conventional care. Outcomes included scores on the 36-item Short Form (SF-36) health survey and the Health Assessment Questionnaire (HAQ) and its disease-specific symptom scales. Differences in scores between the groups were compared using linear models, adjusting for baseline scores and inverse probability of treatment and censoring weights. In total, 41 SSc patients who underwent autologous HSCT and 65 SSc patients treated with conventional care were compared. In marginal linear weighted models, the SF-36 physical component summary score was a mean ± SEM 7.02 ± 1.94 points higher at the first annual visit (P = 0.001) and 14.40 ± 6.16 points higher at the seventh annual visit (P = 0.03) in patients treated with autologous HSCT compared to the conventional care group. HAQ scores were significantly better in the autologous HSCT group compared to the conventional care group during follow-up (mean ± SEM difference from baseline -0.57 ± 0.13 [P < 0.001] at the first annual visit and -0.94 ± 0.49 [P = 0.07] at the seventh annual visit). There were no differences in the SF-36 mental component summary scores between the 2 groups either at baseline or during follow-up. This study provides robust complementary HRQoL data, including overall and event-free survival data, to expand on the standard repertoire of biomedical variables, thus potentially supporting the physical benefits of autologous HSCT in patients with SSc.

Identifiants

pubmed: 32909693
doi: 10.1002/art.41519
doi:

Substances chimiques

Immunosuppressive Agents 0

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

305-314

Subventions

Organisme : CIHR
Pays : Canada

Informations de copyright

© 2020, American College of Rheumatology.

Références

Denton CP, Khanna D. Systemic sclerosis. Lancet 2017;390:1685-99.
Domsic RT, Nihtyanova SI, Wisniewski SR, Fine MJ, Lucas M, Kwoh CK, et al. Derivation and external validation of a prediction rule for five-year mortality in patients with early diffuse cutaneous systemic sclerosis. Arthritis Rheumatol 2016;68:993-1003.
Hao Y, Hudson M, Baron M, Carreira P, Stevens W, Rabusa C, et al. Early mortality in a multinational systemic sclerosis inception cohort. Arthritis Rheumatol 2017;69:1067-77.
Hudson M, Steele R, Lu Y, Thombs BD, Panopalis P, Baron M. Clinical correlates of self-reported physical health status in systemic sclerosis. J Rheumatol 2009;36:1226-9.
Volkmann ER, Tashkin DP, Sim M, Li N, Goldmuntz E, Keyes-Elstein L, et al. Short-term progression of interstitial lung disease in systemic sclerosis predicts long-term survival in two independent clinical trial cohorts. Ann Rheum Dis 2019;7:122-30.
Herrick AL, Pan X, Peytrignet S, Lunt M, Hesselstrand R, Mouthon L, et al. Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS). Ann Rheum Dis 2017;76:1207-18.
Farge D, Marolleau JP, Zohar S, Marjanovic Z, Cabane J, Mounier N, et al. Autologous bone marrow transplantation in the treatment of refractory systemic sclerosis: early results from a French multicentre phase I-II study. Br J Haematol 2002;119:726-39.
Vonk MC, Marjanovic Z, van den Hoogen FH, Zohar S, Schattenberg AV, Fibbe WE, et al. Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis. Ann Rheum Dis 2008;67:98-104.
Burt RK, Shah SJ, Dill K, Grant T, Gheorghiade M, Schroeder J, et al. Autologous non-myeloablative haemopoeitic stem-cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomized phase 2 trial. Lancet 2011;378:498-506.
Van Laar JM, Farge D, Sont JK, Naraghi K, Marjanovic Z, Larghero J, et al. Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial. JAMA 2014;311:2490-8.
Sullivan KM, Goldmuntz EA, Keyes-Elstein L, McSweeney PA, Pinckney A, Welch B, et al. Myeloablative autologous stem-cell transplantation for severe scleroderma. N Engl J Med 2018;378:35-47.
Atkins HL, Muraro PA, van Laar JM, Pavletic SZ. Autologous hematopoietic stem cell transplantation for autoimmune disease-is it now ready for prime time? Biol Blood Marrow Transplant 2012;18 Suppl:S177-83.
Snowden JA, Saccardi R, Allez M, Ardizzone S, Arnold R, Cervera R, et al. Haematopoietic SCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant 2012;47:770-90.
Sullivan KM, Majhail NS, Bredeson C, Carpenter PA, Chatterjee S, Crofford LJ, et al. Systemic sclerosis as an indication for autologous hematopoietic cell transplantation: position statement from the American Society for Blood and Marrow Transplantation. Biol Blood Marrow Transplant 2018;24:1961-4.
Farge D, Burt RK, Oliveira MC, Mousseaux E, Rovira M, Marjanovic Z, et al. Cardiopulmonary assessment of patients with systemic sclerosis for hematopoietic stem cell transplantation: recommendations from the European Society for Blood and Marrow Transplantation Autoimmune Diseases Working Party and collaborating partners. Bone Marrow Transplant 2017;52:1495-503.
Thombs B, Jewett L, Assassi S, Baron M, Bartlett S, Maia A, et al. New directions for patient-centred care in scleroderma: the Scleroderma Patient-centred Intervention Network (SPIN). Clin Exp Rheumatol 2011;30:S23-9.
Burt RK, Farge D. Autologous HSCT is efficacious, but can we make it safer? Nat Rev Rheumatol 2018;14:189-91.
Puyade M, Maltez N, Lansiaux P, Pugnet G, Roblot P, Colmegna I, et al. Health-related quality of life in systemic sclerosis before and after autologous haematopoietic stem cell transplant: a systematic review. Rheumatology (Oxford) 2019;59:779-89.
Subcommittee for Scleroderma Criteria of the American Rheumatism Association Diagnostic and Therapeutic Criteria Committee. Preliminary criteria for the classification of systemic sclerosis (scleroderma). Arthritis Rheum 1980;23:581-90.
Khanna D, Furst DE, Clements PJ, Allanore Y, Baron M, Czirjak L, et al. Standardization of the modified Rodnan skin score for use in clinical trials of systemic sclerosis. J Scleroderma Relat Disord 2017;2:11-8.
Steele R, Hudson M, Lo E, Baron M, on behalf ofthe Canadian Scleroderma Research Group. Clinical decision rule to predict the presence of interstitial lung disease in systemic sclerosis. Arthritis Care Res (Hoboken) 2012;64:519-24.
Ware J. User’s manual for the SF-36v2 health survey: with a supplement documenting SF-12 health survey. 2nd ed. Lincoln (RI): QualityMetric, Inc.; 2007.
Georges C, Chassany O, Mouthon L, Tiev K, Marjanovic Z, Meyer O, et al. Évaluation de la qualité de vie par le MOS-SF36 dans la sclérodermie systémique. Rev Méd Interne 2004;25:16-21.
Hudson M, Thombs BD, Steele R, Panopalis P, Newton E, Baron M, on behalf of the Canadian Scleroderma Research Group. Health-related quality of life in systemic sclerosis: a systematic review. Arthritis Rheum 2009;61:1112-20.
Khanna D, Furst DE, Wong WK, Tsevat J, Clements PJ, Park GS, et al. Reliability, validity, and minimally important differences of the SF-6D in systemic sclerosis. Qual Life Res 2007;16:1083-92.
Kosinski M, Zhao SZ, Dedhiya S, Osterhaus JT, Ware JE Jr. Determining minimally important changes in generic and disease-specific health-related quality of life questionnaires in clinical trials of rheumatoid arthritis. Arthritis Rheum 2000;43:1478-87.
Pope J. Measures of systemic sclerosis (scleroderma): Health Assessment Questionnaire (HAQ) and Scleroderma HAQ (SHAQ), physician- and patient-rated global assessments, Symptom Burden Index (SBI), University of California, Los Angeles, Scleroderma Clinical Trials Consortium Gastrointestinal Scale (UCLA SCTC GIT) 2.0, Baseline Dyspnea Index (BDI) and Transition Dyspnea Index (TDI) (Mahler's Index), Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR), and Raynaud's Condition Score (RCS). Arthritis Care Res (Hoboken) 2011;63 Suppl 11:S98-111.
Georges C, Chassany O, Mouthon L, Tiev K, Toledano C, Meyer O, et al. Validation of French version of the Scleroderma Health Assessment Questionnaire (SSc HAQ). Clin Rheumatol 2005;24:3-10.
Steen VD, Medsger TA Jr. The value of the Health Assessment Questionnaire and special patient-generated scales to demonstrate change in systemic sclerosis patients over time. Arthritis Rheum 1997;40:1984-91.
Butler EA, Baron M, Fogo AB, Frech T, Ghossein C, Hachulla E, et al. Generation of a core set of items to develop classification criteria for scleroderma renal crisis using consensus methodology. Arthritis Rheumatol 2019;71:964-71.
Hudson M, Thombs B, Baron M. Are the Short Form 36 Physical and Mental Component Summary scores useful measures in patients with systemic sclerosis? Comment on the article by Rannou et al [letter]. Arthritis Rheum 2007;57:1339-40.

Auteurs

Nancy Maltez (N)

The Ottawa Hospital, Ottawa, Ontario, Canada.

Mathieu Puyade (M)

CHU de Poitiers, CIC 1402, Poitiers, France.

Mianbo Wang (M)

Lady Davis Institute, Jewish General Hospital, Montreal, Quebec, Canada.

Pauline Lansiaux (P)

Centre de Référence des Maladies Auto-Immunes Systémiques Rares d'Ile-de-France, AP-HP, Hôpital St. Louis, Université de Paris, Institut de Recherche St. Louis, EA 3518, Paris, France.

Zora Marjanovic (Z)

Hôpital St. Antoine, Paris, France.

Catney Charles (C)

Centre de Référence des Maladies Auto-Immunes Systémiques Rares d'Ile-de-France, AP-HP, Hôpital St. Louis, Université de Paris, Institut de Recherche St. Louis, EA 3518, Paris, France.

Russell Steele (R)

McGill University, Montreal, Quebec, Canada.

Murray Baron (M)

Jewish General Hospital and McGill University Health Center, Montreal, Quebec, Canada.

Ines Colmegna (I)

Jewish General Hospital and McGill University Health Center, Montreal, Quebec, Canada.

Marie Hudson (M)

Lady Davis Institute, Jewish General Hospital, and McGill University, Montreal, Quebec, Canada.

Dominique Farge (D)

Centre de Référence des Maladies Auto-Immunes Systémiques Rares d'Ile-de-France, AP-HP, Hôpital St. Louis, Université de Paris, Institut de Recherche St. Louis, EA 3518, Hôpital St. Antoine, Paris, France, and McGill University, Montreal, Quebec, Canada.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH