OxandroloneTo evaluate the effectiveness of oxandrolone in improving the nutritional status and linear growth of pediatric patients with cystic fibrosis CF. Oxandrolone height records of patients with CF treated with oxandrolone were oxandrolone height for height z score, height velocity HVBMI z score, weight velocity WVTanner 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. No adverse events were reported. Larger studies are trenbolone face bloat to determine if oxandrolone is an effective, safe, and oxandrolone height option to stimulate oxandrolone height, improve weight gain, and promote linear growth in patients with CF.
Oxandrolone Improves Height Velocity and BMI in Patients with Cystic Fibrosis
To evaluate the effectiveness of oxandrolone in improving the nutritional status and linear growth of pediatric patients with cystic fibrosis CF. 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. No adverse events were reported.
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. Cystic fibrosis CF is an autosomal recessive disorder that features chronic pulmonary disease, pancreatic insufficiency, and other organ involvement.
Patients with CF often have malnutrition and short stature. Malnutrition and poor growth velocity are associated with declining pulmonary function, morbidity, and mortality [ 2 — 5 ]. Steady improvements in nutritional status are associated with greater pulmonary function [ 6 ], and evidence-based practice recommendations strongly advise close monitoring of growth and nutritional status as well as aggressive nutritional intervention for patients with CF [ 7 ].
Multiple agents have been used to stimulate appetite and growth in patients with CF, but their utility has been limited due to side effects such as adrenal suppression with megestrol or cost such as growth hormone. Oxandrolone is a relatively weak androgen that stimulates appetite and promotes linear growth and has been safely used to treat a variety of conditions associated with wasting, poor weight gain, or short stature [ 8 ].
Furthermore, oxandrolone is an attractive option in growing children because it cannot be aromatized to estrogen, and therefore estrogen-dependent advancement of the bone age is minimized.
To our knowledge, there are no reports in the literature of the use of oxandrolone in patients with CF. For the past 4 years, children with CF referred to our endocrine clinic for persistent growth concerns in spite of other nutrition-augmentation strategies have occasionally been treated with oxandrolone. This retrospective analysis evaluates the effectiveness of oxandrolone in improving the weight gain and linear growth of these patients.
We reviewed the medical records of all patients in our endocrine clinic with CF who were treated with oxandrolone for at least 8 months between January of and October of Additional information obtained included age, Tanner stage, pulmonary function, liver enzyme levels, and any reported adverse events. This retrospective chart review was approved by the University of Wisconsin Human Subjects Committee. There were no predetermined nutritional cutoffs or pubertal stage for the initiation of oxandrolone.
All subjects were treated with 2. Height was measured on a wall-mounted stadiometer to the nearest 0. Weight was measured on a calibrated beam balance platform scale to the nearest 0. Weight and height measurements were used to calculate the BMI z score, which is the preferred method as opposed to percentage of ideal body weight for monitoring nutritional status in patients with cystic fibrosis [ 7 , 9 , 10 ]. Lung function parameters were measured with a Jaeger Masterscreen spirometer in accordance with the American Thoracic Society guidelines [ 11 ].
Forced vital capacity FVC and forced expiratory volume in one second FEV1 were determined and the results expressed as percentages of the reference values of Wang et al. Data were compared before pre-Ox and after Ox initiation of oxandrolone using a paired t -test.
Five subjects 3 boys and 2 girls were identified. The Table 1 lists the baseline characteristics for each subject. The subjects ranged from 8. All subjects were prescribed oral oxandrolone 2. All subjects had pancreatic insufficiency. At the start of oxandrolone therapy, four subjects had Tanner 1 genitalia or breasts, and one male patient had Tanner 4 genitalia. As a comparison, we obtained data for patients between the ages of 11 and 14 seen between January 1, and December 31, in our CF Clinic.
Figure 1 illustrates the anthropometric changes seen with oxandrolone. When the subject with Tanner 4 genitalia 3 was removed from the analysis, there was still a significant improvement in HV and BMI z score. There was no significant change in pulmonary function or liver enzyme levels, and no adverse events were reported.
Neither female subject experienced hirsutism, clitoromegaly, or any evidence of hyperandrogenism. One female subject 2 remained on oxandrolone for a total of 38 months beyond the time frame used to calculate the anthropometric data shown above and had normal pubertal progression and menarche.
One subject 4 had CF-related liver disease with mildly elevated liver enzyme tests at the initiation of oxandrolone, and his liver enzyme tests improved slightly while on oxandrolone.
Two subjects 4 and 5 had previously been on mirtazapine to stimulate appetite. In this brief clinical report, we describe 5 children with CF who received oxandrolone for at least 8—12 months.
All 5 children had height z scores well below the clinic average, and 3 of the subjects had BMI z scores below the clinic average. Oxandrolone improved the height velocity and BMI z score in these patients with CF and showed a trend towards significance in weight velocity and height z score.
No significant changes in pulmonary function or liver enzyme tests were appreciated. Optimizing linear growth and weight gain is critically important in the care of children with CF.
A comprehensive practice recommendation found significant evidence that weight-for-age, height-for-age, and weight-for-height percentiles are associated with improved pulmonary function and survival [ 7 ]. Therefore, increasing weight alone may not fully address the short stature that is commonly seen in patients with CF. Ideally, a growth-promoting agent in patients with CF would improve both weight and height. Multiple appetite stimulants have been studied in patients with CF, including megestrol, cyproheptadine, atypical antipsychotics, and antidepressants [ 15 ].
Megestrol has been shown to improve weight in patients with CF, but side effects include adrenal suppression, glucose intolerance, and diabetes [ 16 — 18 ]. More importantly, the nonanabolic glucocorticoid actions of megestrol promote accumulation of fat mass in excess of lean body mass. In order to stimulate appetite but to also improve body composition, anabolic agents have been used in patients with CF.
Growth hormone GH is a potent anabolic agent that has the capacity to improve the nutritional status of patients with CF. Several studies have shown that GH in children with CF leads to improved height and weight [ 19 — 23 ], while one study found only an improvement in height [ 24 ]. While there were theoretical concerns of glucose intolerance with the use of GH, no study of GH in patients with CF has described this complication.
There are a few disadvantages of GH. A minor concern is the use of daily injections, while a major concern is cost. Insulin is an anabolic hormone that can improve BMI in patients with CF-related diabetes, but insulin has not been studied in patients without CF-related diabetes and carries the obvious risk of hypoglycemia.
Several articles from the s describe the use of older anabolic steroids stanozolol, methandrostenolone, and norethandrolone in patients with CF [ 25 — 27 ]. Oxandrolone is a weak oral androgen taken once daily that has marked anabolic properties with minimal androgenic effects [ 28 ]. Oxandrolone is clinically efficacious across a variety of diseases associated with catabolism and wasting and is FDA-approved as adjunctive therapy for weight loss due to catabolic conditions in both adults and children [ 8 ].
Oxandrolone has also been used as a height-promoting agent. Prior to the availability of recombinant human GH, oxandrolone was commonly used to increase growth velocity in girls with Turner Syndrome [ 29 , 30 ]. Oxandrolone has also been shown to increase growth velocity but not eventual adult height in boys with constitutional delay of growth and puberty [ 31 ]. Oxandrolone cannot be aromatized to estrogen, which minimizes advancement of the bone age. Oxandrolone is also relatively cheap.
The average wholesale price of a 2. While oxandrolone is generally well-tolerated, there are important potential side effects to consider. Oxandrolone can cause transient elevations in liver transaminases [ 8 ], which could be a factor in patients with or at risk for CF-related liver disease. Importantly, however, oxandrolone does not appear to cause the severe hepatotoxic effects associated with other anabolic steroids [ 8 ]. Secondly, while oxandrolone has a high anabolic to androgenic ratio [ 28 ], girls in particular may experience dose-dependent androgenic side effects, such as increase in body hair, deepened voice, or clitoral hypertrophy [ 30 ].
In a review of the efficacy and safety of oxandrolone used at modest dosages e. Nonetheless, it would be prudent to monitor closely for signs of excess androgen in girls treated with oxandrolone. This retrospective, noncontrolled report of a small number of subjects has intrinsic limitations.
Changes in clinical and anthropometric outcomes may be confounded by the disease itself which waxes and wanes or by early puberty. The duration of the study was also relatively short, which makes it difficult to determine if oxandrolone had a positive effect on pulmonary function tests or a negative effect on liver enzyme tests. In this small retrospective exploratory study, oxandrolone safely and effectively improved the height velocity and BMI z score in patients with CF.
Larger prospective studies are needed to determine more conclusively whether oxandrolone should be considered an effective, safe, and affordable option to stimulate appetite and promote growth in patients with CF.
National Center for Biotechnology Information , U. Int J Pediatr Endocrinol. Published online Jan Seffrood , 2 Ellen L. Connor , 1 Michael J. Rock , 1 and David B. Received Sep 4; Accepted Nov 3. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
This article has been cited by other articles in PMC. Introduction Cystic fibrosis CF is an autosomal recessive disorder that features chronic pulmonary disease, pancreatic insufficiency, and other organ involvement.
Materials and Methods We reviewed the medical records of all patients in our endocrine clinic with CF who were treated with oxandrolone for at least 8 months between January of and October of Results Five subjects 3 boys and 2 girls were identified.
Table 1 Patient characteristics at the initiation of oxandrolone treatment. Open in a separate window. Discussion In this brief clinical report, we describe 5 children with CF who received oxandrolone for at least 8—12 months. Conclusion In this small retrospective exploratory study, oxandrolone safely and effectively improved the height velocity and BMI z score in patients with CF. Annual Data Report to the Center Directors. Cystic Fibrosis Foundation; Wasting as an independent predictor of mortality in patients with cystic fibrosis.
A comparison of survival, growth, and pulmonary function in patients with cystic fibrosis in Boston and Toronto. Journal of Clinical Epidemiology.