Nutritional practices at a glance: spinal muscular atrophy type I nutrition survey findings.

Proactive nutritional management for children with spinal muscular atrophy type I can provide insight into improved spinal muscular atrophy care. This observational study consisted of a nutritional and medical history survey of children with spinal muscular atrophy type I collected in 2009-2011. Forty-four caregiver survey responses were evaluated using descriptive statistics. Average age of spinal muscular atrophy type I subjects was 5 years (5 mo-16 y). The subject cohort was composed of 22 males, 21 females, and 1 unreported. Nutrition support via feeding tube was utilized by 43 of 44 subjects. A majority of respondents reported using elemental or semi-elemental formula for subjects' essential caloric intake (34 of 44). Formula intolerance issues were reported by many caregivers (27 of 44). Half of caregivers implemented dietary changes on their own or with guidance from other families; 15 caregivers consulted a registered dietitian. Survey responses and comments indicate need for evidence-based nutritional guidelines for spinal muscular atrophy.

Oxandrolone Improves Height Velocity and BMI in Patients with Cystic Fibrosis.

Objective. To evaluate the effectiveness of oxandrolone in improving the nutritional status and linear growth of pediatric patients with cystic fibrosis (CF). Methods. Medical records of patients with CF treated with oxandrolone were reviewed for height z score, height velocity (HV), BMI z score, weight velocity (WV), Tanner stage, pulmonary function, liver enzyme levels, and any reported adverse events. Data were compared before (pre-Ox) and after (Ox) oxandrolone using a paired t-test. Results. 5 subjects (ages 8.5-14.5 years) were treated with oxandrolone 2.5 mg daily for 8-38 months. After 8-12 months of treatment, there was a statistically significant improvement in HV (pre-Ox = 5.3 +/- 1.4 cm/yr, Ox = 8.3 +/- 1.2 cm/yr, P < .01) and BMI z score (pre-Ox = -0.61 +/- 1.04, Ox = -0.30 +/- 0.86, P = .02). Both height z score (pre-Ox = -1.64 +/- 0.63, Ox = -1.30 +/- 0.49, P = .057) and WV (pre-Ox = 4.2 +/- 3.7 kg/yr, Ox = 6.8 +/- 1.0 kg/yr, P = .072) showed beneficial trends that did not reach statistical significance. No adverse events were reported. Conclusions. In this brief clinical report, oxandrolone improved the HV and BMI z score in patients with CF. Larger studies are needed to determine if oxandrolone is an effective, safe, and affordable option to stimulate appetite, improve weight gain, and promote linear growth in patients with CF.