Growth after recombinant human growth hormone (rhGH) treatment in transplanted thalassemic patients

Abstract

Summary:(BU) and cyclophosphamide (CY) can cure a high percentage of patients with compatible donors. Little is known about the long-term effects of BMT in patients with thalasThe aim of this study was to evaluate the treatment effects with recombinant human growth hormone semia in relation to the growth process as well as endocrine function. (rhGH) in a group of patients after bone marrow transplantation for thalassemia major. At the end of treat-In the present study we have analysed the treatment effects with recombinant human growth hormone (rhGH) ment we divided the subjects into two groups according to the outcome of the therapy: responder and nonin patients after BMT for thalassemia major. responder. Responder group: after 24 months of rhGH administration, growth rate was still significantly higher in respect to start of treatment (P Ͻ 0.0001). Plasma lev- Patients and methods els of IGF-I rose significantly (P Ͻ 0.003). The serum levels of serum asparate aminotransferase (SGOT) andThe study group comprised seven patients (five males, two females) ranging in age from 11.7 to 12.7 years alanine aminotransferase (SGPT) were higher compared to normal values but improved in non-responder (12.06 ± 0.76), affected by thalassemia major and who underwent BMT (mean age at BMT was 7.11 ± 3.31 years) patients. There was no difference in the mean concentration of these parameters before and after treatment in Pescara, Italy. Prior to the referral for BMT all patients had received multiple erythrocyte transfusions. The median (P = NS). Non-responder group: these patients had a worsening of the growth rate during rhGH adminisnumber of tranfusions was 114.71 ± 57.67 (range 13-180). They were administered daily doses of 0.9 IU/kg body tration. There was no increase of the IGF-I levels. Single values of transaminase and ferritin levels were higher weight per week subcutaneously of rhGH (SAIZEN Serono, Rome, Italy) for 24 consecutive months. than in responder patients before and after treatment. There was a significant correlation between IGF-I, Assessment of height, other auxologic parameters and laboratory measures were performed before treatment and SGOT, SGPT and ferritin in all patients before and after therapy. It appears from these data that rhGH at 6 monthly intervals during GH therapy.At the end of treatment we divided the subjects into two administration is worth serious consideration in patients after BMT for thalassemia major presenting impaired groups according to the outcome of the therapy: responder (four boys, one girl) and non-responder (one boy, one girl) growth hormone secretion. This treatment can offer good results only in cases where the normal hepatic synpatients whose height velocity increase was more than 2 cm/year above baseline or less, respectively. thesis of IGF-I is conserved and where liver damage has not reached irreversible conditions, as we have seen inInformed consent for the study was obtained from the patients' parents. the responder group.

Final height of thalassemic patients who underwent bone marrow transplantation during childhood

“…24 Many studies dealing with the growth of these patients who have undergone bone marrow transplantation report only limited data for the first few years after transplantation. 24,25 In this study we analyzed the final height achieved by a population of patients with thalassaemia major who underwent allogeneic bone marrow transplantation during childhood. None of the patients had GH deficiency.…”

Endocrine late effects after bone marrow transplant

“…In a subset of patients transplanted for thalassaemia, good responders to GH therapy over the first 2 years appeared to be those in whom the normal hepatic synthesis of IGF‐1 was conserved, and liver damage was not irreversible (De Simone et al , 1997); the latter study was short‐term and contained very small numbers of patients.…”

Association of gingival crevicular fluid and serum intracytoplasmic enzyme levels in periodontally healthy homozygous (major) β‐thalassemia patients

“…LDH and AST have been used in assessing tissue destruction both in periodontal disease (Eley & Cox 1998) and thalassemia major (Hoffbrand et al 1979, Wolfe et al 1985, Triadou et al 1989, Di Marco et al 1993, Toren et al 1996, De Simone et al 1997.…”

“…Serum LDH is mildly elevated because of ineffective erythropoiesis and has been used to monitor intramedullary hemolysis (Toren et al 1996). Serum AST and ALT have been employed to evaluate the level of hepatic and cardiac functions (Hoffbrand et al 1979, Wolfe et al 1985, Triadou et al 1989, Di Marco et al 1993, De Simone et al 1997.…”