Aumento do crescimento de pacientes com Duchenne em tratamento com corticóides e tratados com GH (hormônio do crescimento)

USA - esta pesquisa foi apresentada no congresso anual da Sociedade de Endocrinologia - ENDO-2010. Há muitas controvérsias com relação ao uso de hormônio do crescimento (GH) em distrofia muscular de Duchenne. O GH poderia aumentar o crescimento de adolescentes em uso de corticóides e poderia ter outros efeitos positivos na função muscular e na gordura corpórea; 29 adolescentes com Duchenne em tratamento com corticóides foram tratados com GH por 4 a 32 meses. Houve um aumento do crescimento, com redução da velocidade de ganho de peso sem alterações colaterais significativas; um menino teve resistência à insulina e dois progressão da escoliose. Os estudos devem prosseguir por mais tempo para conclusões definitivas.

O resumo em inglês pode ser lido abaixo:

(Annual Meeting Endocrinology Society - ENDO-2010) Growth Hormone Improves Growth in Duchenne Muscular Dystrophy Boys with Steroid-Induced Growth Failure.

MM Rutter, J Collins, JG Woo, SR Rose, H Sawnani, LH Cripe, KJ Kinnett, K Hor, BL Wong. Cincinnati Children's Hosp Med Ctr, Cincinnati, OH.

Background: Duchenne Muscular Dystrophy (DMD) is a progressive degenerative muscle disorder affecting 1 in 3500 boys. In the absence of a cure, daily high-dose glucocorticoids (GCs) are the mainstay of treatment, slowing disease progression and prolonging ambulation and survival. GCs cause growth failure, weight gain, absent puberty and osteoporosis, which negatively impact quality of life in DMD. Growth hormone (GH) offers potential benefit in DMD: it may help counter GC-induced growth failure, and could have positive effects on body fat, muscle strength and function. However, data regarding efficacy and safety of GH in DMD is lacking.
Objective: To evaluate efficacy and safety of GH in DMD boys with GC-induced growth failure during the first year of treatment.
Methods: We report a case-series of 29 DMD boys on daily GCs, treated in the Cincinnati Neuromuscular Comprehensive Care Center. The boys were treated with growth hormone for severe growth failure. Outcomes included growth velocity, height SD, weight, BMI, neuromuscular and cardiopulmonary function, and side effects.
Results: 29 prepubertal boys (mean age SD 12.2 2.9y) were treated with GH for 4-32m (mean 12m). They had received daily GCs for 5.5 2.2y. Peak stimulated GH levels were 6.9 3.6 ng/ml. Height z-score (mean SEM) was -3.1 0.2 and height velocity was 1.1 0.3 cm/y before GH. During the first year on GH, height velocity improved to 5.6 0.7 cm/y (p<0.0001). Baseline decline in height z-score before GH (p<0.001) was followed by stabilization at -3.0 0.2 (p=0.2) on GH. There was a trend toward reduction in rate of weight gain at 1 year (2.8 0.7 to 0.6 1.1 kg/y, p=0.3), with decreased weight z-scores from -0.6 0.3 to -1.2 0.4 (p<0.0004). BMI z-score improved from 1.3 0.2 to 0.8 0.2 (p<0.0001). There were no detrimental effects on neuromuscular or cardiopulmonary function attributable to GH. GH was well tolerated, with 3/29 experiencing side effects by 1y. One boy developed worsening insulin resistance / impaired glucose tolerance. Two boys had progression of scoliosis.
Conclusions: GH treatment of DMD boys with GC-induced growth failure improved growth during the first year. Rate of weight gain slowed for some, with improvement in BMI. GH was relatively safe, with no detrimental effects on neuromuscular and cardiopulmonary function. Further study is needed before conclusions can be drawn regarding longer-term safety and efficacy.

Fonte: http://distrofiamuscular.net/noticias.htm