Somatropin treatment of spinal muscular atrophy: a placebo-controlled, double-blind crossover pilot study.

In preclinical studies growth hormone and its primary mediator IGF-1 have shown potential to increase muscle mass and strength. A single patient with spinal muscular atrophy reported benefit after compassionate use of growth hormone. Therefore we evaluated the efficacy and safety of growth hormone treatment for spinal muscular atrophy in a multicenter, randomised, double-blind, placebo-controlled, crossover pilot trial. Patients (n = 19) with type II/III spinal muscular atrophy were randomised to receive either somatropin (0.03 mg/kg/day) or placebo subcutaneously for 3 months, followed by a 2-month wash-out phase before 3 months of treatment with the contrary remedy. Changes in upper limb muscle strength (megascore for elbow flexion and hand-grip in Newton) were assessed by hand-held myometry as the primary measure of outcome. Secondary outcome measures included lower limb muscle strength, motor function using the Hammersmith Functional Motor Scale and other functional tests for motor function and pulmonary function. Somatropin treatment did not significantly affect upper limb muscle strength (point estimate mean: 0.08 N, 95% confidence interval (CI:-3.79;3.95, p = 0.965), lower limb muscle strength (point estimate mean: 2.23 N, CI:-2.19;6.63, p = 0.302) or muscle and pulmonary function. Side effects occurring during somatropin treatment corresponded with well-known side effects of growth hormone substitution in patients with growth hormone deficiency. In this pilot study, growth hormone treatment did not improve muscle strength or function in patients with spinal muscular atrophy type II/III.

Transvaginal color Doppler sonography and conventional sonography in the preoperative assessment of adnexal masses.

OBJECTIVE: This investigation was undertaken to evaluate the predictive value of the blood flow velocity as measured by transvaginal color Doppler sonography (CDS) and conventional sonographic criteria in the preoperative assessment of ovarian tumors. METHODS: 98 patients who were admitted for surgery with a suspicion of an adnexal mass (69 benign tumors, 4 borderline tumors, and 25 malignant tumors) were prospectively studied with transvaginal sonography (TVS) and CDS. Pulsatility index of blood flow velocity waveforms was evaluated by CDS and compared with established sonographic criteria for discriminating between benign and malignant ovarian tumors. RESULTS: TVS had a sensitivity of 91% and a specificity of 84% in detecting malignant ovarian tumors compared with CDS with a sensitivity of 90% and a specificity of 74%, using 1.1 as a cut-off value for the pulsatility index. The specificity of CDS was higher in postmenopausal (88%) than in premenopausal (63%) patients. Using TVS and CDS combined, the sensitivity and specificity of diagnosing malignant ovarian tumors could be increased to 95% and 86%. CONCLUSION: In premenopausal patients, TVS is clearly a more sensitive and more specific technique than CDS in correctly identifying benign and malignant ovarian tumors, whereas in postmenopausal patients, CDS gives useful additional information concerning blood vessel resistance. If CDS is used as an additional technique to TVS, it can increase the sensitivity and specificity of TVS in discriminating between benign and malignant ovarian tumors.