Michelle: I have doubts about the indications of growth hormone in DMD. There are fews association with nanism (GH deficiency) and DMD but in 2 cases the use of GH improve the symptoms. One description:
1)Duchenne muscular dystrophy with associated growth hormone deficiency
Ghafoor T, Mahmood A, Shams S.
Department of Paediatrics, PNS Shifa, Karachi. drghafoortariq@hotmail.com
A patient with Duchenne muscular dystrophy (DMD) and growth hormone (GH) deficiency is described who had no clinical evidence of muscular weakness before initiation of GH replacement therapy. Treatment with human GH resulted in appearance of symptoms of easy fatigability and proximal muscle weakness. Thorough investigations including serum creatinine phosphokinase (CK) levels is recommended in every patient with GH deficiency before starting GH replacement therapy.
www.ncbi.nlm.nih.gov/pubmed/15569563?ordinalpos=4&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_RVDocSumOld studies tried the possibility to slow the evolution of DMD with mazindol, a bad GH inhibitor without success:
Randomized, double-blind trial of mazindol in Duchenne dystrophy.
Griggs RC, Moxley RT 3rd, Mendell JR, Fenichel GM, Brooke MH, Miller PJ, Mandel S, Florence J, Schierbecker J, Kaiser KK, et al.
Department of Neurology, University of Rochester, New York.
There is evidence that growth hormone may be related to the progression of weakness in Duchenne dystrophy. We conducted a 12-month controlled trial of mazindol, a putative growth hormone secretion inhibitor, in 83 boys with Duchenne dystrophy. Muscle strength, contractures, functional ability and pulmonary function were tested at baseline, and 6 and 12 months after treatment with mazindol (3 mg/d) or placebo. The study was designed to have a power of greater than 0.90 to detect a slowing to 25% of the expected rate of progression of weakness at P less than 0.05. Mazindol did not benefit strength at any point in the study. Side effects attributable to mazindol included decreased appetite (36%), dry mouth (10%), behavioral change (22%), and gastrointestinal symptoms (18%); mazindol dosage was reduced in 43% of patients. The effect of mazindol on GH secretion was estimated indirectly by comparing the postabsorptive IGF-I levels obtained following 3, 6, 9, and 12 months in the mazindol treated to those in the placebo groups. Although mazindol-treated patients gained less weight and height than placebo-treated patients, no significant effect on IGF-I levels was observed. Mazindol doses not slow the progression of weakness in Duchenne dystrophy.
www.ncbi.nlm.nih.gov/pubmed/2266990?ordinalpos=24&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_RVDocSumBut GH can increase Igf 1 and can give some good effect in dmd:
A preliminary randomized study of growth hormone administration in Becker and Duchenne muscular dystrophies.Cittadini A, Ines Comi L, Longobardi S, Rocco Petretta V, Casaburi C, Passamano L, Merola B, Durante-Mangoni E, Saccà L, Politano L.
Department of Internal Medicine and Cardiovascular Sciences, University Federico II, Naples, Italy. cittadin@unina.it
AIM: Since growth hormone (GH) has proven beneficial in experimental heart failure, and the natural history of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) is frequently complicated by the development of dilated cardiomyopathy, we administered GH to six patients with DMD and 10 with BMD, with the evidence of cardiac involvement. METHODS AND RESULTS: Patients were randomized to receive for 3 months either placebo or recombinant human GH, in a double-blind fashion. In GH-treated patients, left ventricular (LV) mass increased by 16% in BMD and by 29% in DMD (both p<0.01), with a significant increase of relative wall thickness (+19%). Systemic blood pressure remained unchanged, while LV end-systolic stress fell significantly by 13% in BMD and by 33% in DMD, with a slight increase of systolic function indexes. No changes were observed related to cardiac arrhythmias and skeletal muscle function in the patient groups during the treatment period, nor any side effects were observed. Brain natriuretic peptide, interleukin-6, and tumor necrosis factor-alpha circulating levels were elevated at baseline. While brain natriuretic peptide decreased by 40%, cytokine levels did not exhibit significant variations during the treatment period. CONCLUSIONS: The 3-month GH therapy in patients with DMD and BMD induces a hypertrophic response associated with a significant reduction of brain natriuretic peptide plasma levels and a slight improvement of systolic function, no changes in skeletal muscle function, and no side effects.
www.ncbi.nlm.nih.gov/pubmed/12657225?ordinalpos=9&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_RVDocSumPotential for growth factor treatment of muscle disease.Moxley RT 3rd.
University of Rochester School of Medicine, Department of Neurology, NY 14642.
The recent development of recombinant human growth hormone (GH) and insulin-like growth factor 1 (IGF-1) has provided the opportunity to study the effects of these growth factors in humans. The majority of studies have been performed in healthy young and older adults. Both GH and IGF-1 have shown very positive effects on muscle protein anabolism. Relatively few side effects have developed. Initial studies have shown a beneficial effect of GH in reversing the catabolic actions of oral prednisone, and GH treatment has led to an increase in muscle mass and protein synthesis in pilot studies of myotonic dystrophy. Combination therapy in normal subjects using both GH and IGF-1 has shown encouraging results. There is a greater enhancement of protein anabolism during combined treatment than there is with either agent alone, and concomitant therapy with GH diminished the side effects of IGF-1. It is now feasible to consider therapeutic trials in patients. Future studies of GH and IGF-1 treatment in patients with primary muscle disease and in patients receiving long-term corticosteroid therapy are needed.
www.ncbi.nlm.nih.gov/pubmed/7804464?ordinalpos=20&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_RVDocSumHence the use of growth hormone must be well appraised to foresee the good and bad effects of the therapy.
