Prognostic factors and evolution of acquired aplastic anemia in childhood. A prospective analysis of 48 androgen-treated cases

Am J Pediatr Hematol Oncol. 1982 Fall;4(3):273-83.

Prognostic factors and evolution of acquired aplastic anemia in childhood. A
prospective analysis of 48 androgen-treated cases.

Najean Y, Girot R, Baumelou E.

Forty-eight cases of acquired aplastic anemia in children were analyzed in
comparison to 26 cases of genetic aplastic anemia and 483 cases of aplastic
anemia in adults. All were gathered from similar institutions and all were
similarly followed and treated with androgens. The following conclusions were
drawn: 1) Initial severity is greater in children than in adults, and is greater
in acquired than in genetic aplastic anemia; 2) even in cases of similar initial
severity, the early death rate is higher in children than in adults; 3) a
multiparametric index allows the correct prediction of short-term evolution in
70% of the cases and thus aids in providing an indication for bone marrow graft;
its sensitivity is similar to that of the classical parameters proposed by
Camitta, et al., but its specificity significantly higher; 4) most deaths
occurred during the first 3-4 months and the chance for long-term improvement
appears similar in the more severe than in the less severe cases if they survive
this delay; 5) some data (relapse after androgen withdrawal and
androgen-dependence and failure of corticoid therapy alone) suggest that androgen
therapy in children is useful, as it is in adults, and that corticosteroids do
not modify the course of the disease at its usual dosage (1 mg/kg/day); and 6)
very few side effects, particularly concerning height, of androgens were noted in
the survivors at adult age after long-term androgen therapy prescribed before
puberty.