Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy1.

Autor:	McDonald CM; University of California Davis Health, Sacramento, CA, USA., Mayer OH; Children's Hospital of Philadelphia, Philadelphia, PA, USA., Hor KN; Nationwide Children's Hospital, Columbus, OH, USA., Miller D; CureDuchenne, Newport Beach, CA, USA., Goemans N; University of Leuven, Leuven, Belgium., Henricson EK; University of California Davis Health, Sacramento, CA, USA., Marden JR; Analysis Group, Inc., Boston, MA, USA., Freimark J; Analysis Group, Inc., Boston, MA, USA., Lane H; Analysis Group, Inc., Boston, MA, USA., Zhang A; Analysis Group, Inc., Boston, MA, USA., Frean M; Analysis Group, Inc., Boston, MA, USA., Trifillis P; PTC Therapeutics, Inc. South Plainfield, NJ, USA., Koladicz K; PTC Therapeutics, Inc. South Plainfield, NJ, USA., Signorovitch J; Analysis Group, Inc., Boston, MA, USA.
Jazyk:	angličtina
Zdroj:	Journal of neuromuscular diseases [J Neuromuscul Dis] 2023; Vol. 10 (1), pp. 67-79.
DOI:	10.3233/JND-221575
Abstrakt:	Background: Evidence on the long-term efficacy of steroids in Duchenne muscular dystrophy (DMD) after loss of ambulation is limited. Objective: Characterize and compare disease progression by steroid treatment (prednisone, deflazacort, or no steroids) among non-ambulatory boys with DMD. Methods: Disease progression was measured by functional status (Performance of Upper Limb Module for DMD 1.2 [PUL] and Egen Klassifikation Scale Version 2 [EK] scale) and by cardiac and pulmonary function (left ventricular ejection fraction [LVEF], forced vital capacity [FVC] % -predicted, cough peak flow [CPF]). Longitudinal changes in outcomes, progression to key disease milestones, and dosing and body composition metrics were analyzed descriptively and in multivariate models. Results: This longitudinal cohort study included 86 non-ambulatory patients with DMD (mean age 13.4 years; n = 40 [deflazacort], n = 29 [prednisone], n = 17 [no steroids]). Deflazacort use resulted in slower average declines in FVC % -predicted vs. no steroids (+3.73 percentage points/year, p < 0.05). Both steroids were associated with significantly slower average declines in LVEF, improvement in CPF, and slower declines in total PUL score and EK total score vs. no steroids; deflazacort was associated with slower declines in total PUL score vs. prednisone (all p < 0.05). Both steroids also preserved functional abilities considered especially important to quality of life, including the abilities to perform hand-to-mouth function and to turn in bed at night unaided (all p < 0.05 vs. no steroids). Conclusions: Steroid use after loss of ambulation in DMD was associated with delayed progression of important pulmonary, cardiac, and upper extremity functional deficits, suggesting some benefits of deflazacort over prednisone.
Databáze:	MEDLINE
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