Dados do Trabalho

Título

LONG TERM USE OF DEFLAZACORT OR PREDNISOLONE IN PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY: EXPERIENCE AT A LARGE BRAZILIAN CENTER

Introdução

Duchenne muscular dystrophy (DMD) is a severe progressive inherited neuromuscular disorder, caused by mutations in DMD gene. Although onset of disease can be observed during the first age of live, most patients exhibit signs of muscle weakness between 3 to 5years of age and around 10-12 years of age individuals loss ambulation (LoA). Standard care treatment of DMD include the use of steroids. The two most commonly prescribed in Brazil are prednisolone and deflazacort. Use of steroids modified the natural history of the disease by slowing the progression of motor and pulmonary functional decline and extending survival.

Objetivo

Analise data of a group of 118 ambulatory and non-ambulatory Brazilian boys with DMD in steroid treatment followed in service for neuromuscular disorders at our Institution - Hospital das Clinicas of University of São Paulo, Brazil.

Métodos

A retrospective cohort analysis 118 patients with DMD in steroid use who attended our clinic in the last 7 years (from 2016 to 2022). Treatment with steroid, prednisolone on intermittent regimen (10 days on and 10 days off) at a dose of 0,75 mg/kg/day, or deflazacort daily at a dose of 0.9 mg/kg/day was started by decision of first author. The outcomes of interest were age at last visit, age of diagnosis, age at steroid was initiated and age at loss of ambulation.

Resultados

The mean age at last clinic visit was 10,1 years. The age at onset of the disease ranged from 1 to 7 years (mean 3,3 years). The mean age at diagnosis was 7,1 years (range 2-13 years). The mean age at starting treatment with steroid was 7,3 years (range 2-14 years). Deflazacort (70%) is more common used than prednisolone (30%), but 20 patients switched prednisolone to deflazacort during follow-up due to side effects or not enough benefit. 37/118 (35,59%) of boys loss of ambulation. In deflazacort group, LoA occurred by the age of 9.31±2.46 years; and in prednisolone group, LoA was observed at the age of 10.36±1.86 (p >0.05), without statistical significance.

Conclusões

Loss of ambulation (LoA) represents a clinically meaningful milestone in DMD progression. The results of this study showed that in our center the LoA occurred at an earlier age when compared to others studies That may be related to a late diagnosis and treatment. There were no statistical differences between prednisolone or deflazacort use at age of LoA, but weight gain and lack of response to treatment seem to be more evident in patients treated with prednisolone.

Palavras chave

Duchenne, deflazacort, prednisolona, DMD, steroid

Referências (se houver)

Nascimento Osorio A, Medina Cantillo J, Camacho Salas A, Madruga Garrido M, Vilchez Padilla JJ. Consensus on the diagnosis, treatment and follow-up of patients with Duchenne muscular dystrophy. Neurologia (Engl Ed). 2019 Sep;34(7):469-481. English, Spanish. doi: 10.1016/j.nrl.2018.01.001. Epub 2018 Mar 9. PMID: 29526319.

Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010 Jan;9(1):77-93. doi: 10.1016/S1474-4422(09)70271-6. Epub 2009 Nov 27. PMID: 19945913.

Rao VK. Guidelines for Corticosteroid use in Treatment of DMD. Pediatr Neurol Briefs. 2016 Mar;30(3):21. doi: 10.15844/pedneurbriefs-30-3-4. PMID: 27397628; PMCID: PMC4936972.

Fonte de Fomento (se houver)

Declaração de conflito de interesses de TODOS os autores

não ha

Área

Doenças neuromusculares