ANABOLIC STEROIDS

1961 Kunstadter RH, Mendelsohn RS. Norethandrolone in children with and without cystic fibrosis of the pancreas. Illinois M J 1961; 120:156-161. [PubMed]
In 1961 as the chest infection progressed and increased in severity, it was very difficult, and usually impossible, to achieve normal weight gain and growth in children with cystic fibrosis. As the available pancreatic enzyme supplements were relatively inefficient, most patients took a low fat diet to avoid very unpleasant bowel symptoms; also there was a severe catabolic wasting effect from the active and increasingly severe chest infection. However, in this report of 14 children with CF treated with anabolic steroids had “remarkable gains in weight”.

Shwachman commented that he had used one such preparation (Nilevar) on 30 patients and found the drug “very useful but not to be used routinely”. This was the first of a number of reports that anabolic steroids had a favourable effect on weight gain in children with cystic fibrosis. The drugs were to become quite widely used as nutritional problems became increasingly severe as more children survived for longer and nutrition continued as an increasingly significant problem. It was not until the early Eighties that there was marked improvement in the control of the intestinal malabsorption following the introduction of the acid resistant enzymes (Pancrease and later Creon); also around that time, for those with more severe nutritional problems, more aggressive nutritional interventions such as nasogastric and gastrostomy feeds, became available. So the use of anabolic steroids gradually declined (also Dooley RR et al. J Pediatr 1969; 74:95-102. [PubMed] The subject was reviewed in 1981 by Richard Dooley (Anabolic steroids. In 1000 years of Cystic Fibrosis. Warren Warwick (ed). University of Minnesota, 1981).

1966 Good TA. Bessman SP. Anabolic steroids in cystic fibrosis of the pancreas. Am J Dis Child 1966; 111:272-277. [PubMed]

1969 Dooley RR, Moss AJ, Wright PM, Hassakis PC. Norethandrolone in cystic fibrosis of the pancreas. J Pediatr 1969; 74: 95-102. [PubMed]
Twenty eight severely affected patients were treated in the 5 years up to 1968 with the anabolic steroid norethandrolone. All the patients showed immediate improvement in appetite, activity and weight gain; also their respiratory function improved. Six patients died and there were a variety of side effects side effects including virilisation (also Kunstadter et al, 1961 above). However, there was great interest in this treatment as all else had failed to achieve weight gain in some of these patients. The subject was reviewed in 1981 by Richard Dooley (Anabolic steroids. In 1000 years of Cystic Fibrosis. Warren Warwick (ed). University of Minnesota, 1981).

2005 Colombo C. Battezzati A. Growth failure in cystic fibrosis: a true need for anabolic agents?. J Pediatr 2005; 146:303-305. [PubMed]
2009 Varness T. Seffrood EE. Connor EL. Rock MJ. Allen DB. Oxandrolone Improves Height Velocity and BMI in Patients with Cystic Fibrosis. Internat J Pediatr Endocrin 2009:, [PubMed] A retrospective study to evaluate the effectiveness of oxandrolone in improving the nutritional status and linear growth of pediatric patients with cystic fibrosis (CF). Both height z score (pre-Ox = -1.64 +/- 0.63, Ox = -1.30 +/- 0.49, P = .057) and weight velocity (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. The authors concluded oxandrolone improved the HV and BMI z score in patients with CF but larger studies were 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.

There were a number of papers on the use of anabolic steroids before more effective pancreatic enzymes became available (see Topic section anabolic steroids). Although they were effective, the side effects were a problem. Apparently there are fewer androgenic side effects with the newer preparation described in this paper.