Experience with refractory vitamin D-resistant rickets and non-17α alkyl testosterone derivative anabolic agent

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Background:Renal tubular disorders may be associated vitamin D-resistant rickets, dwarfism and deformities. Treatment is nonspecific and directed towards correcting acidosis, hypokalemia, hypophosphatemia and vitamin D resistance. The response to therapy depends on the severity of the disorder, and sometimes bone changes persist with the development of skeletal deformities.Patients & methods:From June 2000 to 2001, four patients with renal tubular disorder associated with vitamin D-resistant rickets and deformities despite previous therapies, were observed. Of these patients, three have hereditary (autosomal recessive) proximal renal tubular acidosis affecting siblings with perfectly normal parents and grandparents, including two sisters, and one patient has X-linked dominant hypophosphatemic rickets. All have at least one sibling who died affected by the same disorder. Two boys, each with proximal renal tubular acidosis and X-linked dominant hypophosphatemic rickets were enrolled in a clinical study investigating the possibility of adding nandrolone to their conventional therapies to promote healing of rickets, provided both were males and having markedly delayed bone age together with close monitoring of bone age and careful observation for any sign of virilization. The boy with X-linked dominant hypophosphatemic rickets aged 18 months at enrollment, continued to have severe active rickets with the development of kyphosis despite 5 months of combined therapy with 1-alfacalcidol and phosphate supplementation. He received seven doses of nandrolone decanoate (12.5 mg) every 4 to 5 weeks. The boy with proximal renal tubular acidosis was aged 7 years at the time of addition of nandrolone to the conventional therapies of alfacalcidol, alkali and potassium supplementation. He also demonstrated severe active rickets and worsening of his bowing deformities. He received ten injections of nandrolone (25 mg) every 2 weeks. Nutritional support was provided for both and their skeletal age was monitored biweekly.Results:The novel addition of nandrolone resulted in a perfect radiological healing of rickets and growth acceleration without the occurrence of unwanted effects or advancement of bone age. The two sisters did not show any clinical or radiological improvement in their rickets and one of them died within 6 months of referral.Conclusion:The judicious use of nandrolone in carefully selected patients with refractory vitamin D-resistant rickets and significant delay of bone age was associated with a beneficial effect on healing of rickets and growth without any obvious adverse effect. This beneficial effect has to be confirmed by additional trials in the future.

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