European Society for Paediatric Research Abstract


Abstract 22 Second part

Second Part

In a phase 3 trial the efficacy of Filgrastim treatment was evaluated as number of hospitalizations and i.v. antibiotic use. These events calculated as EAR (number of events/patients/months) decreased, in a 2.5/3.5 years of treatment, from 15.0 to 5.4 for the hospitalization and from 16.8 to 5.4 for the i.v. antibiotic use. Other clinical events seen in the SCNIR analysis are:

  • thrombocytopenia (< 6% of the cases) in part related to the development of myelodysplastic syndrome or myeloid leukemia and in part to the Filgrastim dosage, which was, however, unrelated to the duration of treatment and recovered after Filgrastim discontinuation

  • the development of MDS/AML, observed in 9.1% of the patients with congenital neutropenia, seems to be related to the type of neutropenia more than to the type and duration of treatment

  • splenomegaly was present in 22% of the congenital group in 13% of the cyclic one and in 8.8% of the idiopathic group. The frequency increase up to 48.8% after a few years of treatment. The spleen size range was 2-4 cm

  • osteopenia/osteoporosis: the frequency of osteopenia-osteoporosis was evaluated in 160 patients out of 531 enrolled in the SCNIR. The data reported through 1997 showed that 41.3% of these patients had varying degrees of abnormal results and that osteopenia-osteoporosis was more frequent in the congenital group

  • vasculitys was present in 2.8% of the cases, was limited to the skin and was related to the number of neutrophils.

Increasing the number of patients enrolled is the main aim of the SCNIR, in order to better understand the pathogenetic mechanisms, the effects of long-term treatment and the genetic aspects of this group of rare disease.

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Mori, P., Freedman, M., Bonilla, M. et al. THE INTERNATIONAL REGISTRY ON SEVERE CHRONIC NEUTROPENIA (SCNIR). Pediatr Res 45, 943 (1999).

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