Background: As a curative therapy, haematopoietic stem cells transplantation (HSCT) has been also used for patients with non-neoplastic diseases such as aplastic anemia, primary immunodeficiency, and some congenital metabolic diseases. For these diseases, the intensity of the conditioning has been reduced comparing to that of malignancy diseases. Therefore, late effects of HSCT for non-neoplastic diseases has been expected to be milder than that for neoplastic diseases, however few studies have been reported and clinical details are not elucidated yet.
Objective and method: Since 19832013, 73 of non-neoplastic disease patients of our institute were received HSCT, and 19 patients were followed up to date (three: severe combined immunodeficiency disease (SCID), two: severe congenital neutropenia, four: Wiskott-Aldrich syndrome (WAS), four: CD40L deficiency, three: aplastic anemia (AA), one each: Chronic Mucocutaneous Candidasis, Ectodermal dysplasia with immunodeficiency, EB virus associated hemophagocytic syndrome). We retrospectively analysed the clinical courses of post-HSCT patients with non-neoplastic diseases.
Results: Because x-linked diseases (SCID, WAS and CD40LD) are main causes of immunodeficiency, the number of males was larger than that of females (16:3). The median age of the patients was 14.0±10.7 (range: 241, median: 12.2), and the mean age at HSCT was 5.9±6.4 (range: 021, median: 3.0). We observed two patients with growth failure whose heights were <−3 S.D., three with hypogonadism and one with subclinical hypothyroidism. Steroid therapy for GVHD could be a risk for short stature. (Fishers exact test, P=0.036), however, other risks for these endocrinological abnormalities, especially gonadal failure were not identified.
Conclusion: Our study revealed HSCT causes late effects even in patients with non-neoplastic disease. In order to clarify the clinical details, further large scaled studies are necessary, and our study suggests the clinical importance of late effects of post HSCT patients with non-neoplastic disease.
01 - 03 Oct 2015
European Society for Paediatric Endocrinology