ESPE Abstracts

Introduction: Growth retardation < –2 SDS is frequently observed in children with chronic kidney disease (CKD) and is a marker of severity of CKD. Recombinant human growth hormone (rhGH) treatment has been approved since 1995 in CKD patients. The aim of this study was to describe the growth outcomes and treatment patterns in children with congenital CKD.

Methods: Patients were recruited from transplantation records in three pediatric academic centers in Ile-de-France. They received a first transplant between 3 and 18 years of age from 2015 to 2020. Patients suffering from cystinosis, a comorbidity or syndromic disease associated with growth failure, patients who had already received a graft and patients transplanted less than 1 year after diagnosis were excluded. Clinical data were collected at 4 time points: diagnosis of the nephropathy, rhGH start, initiation of dialysis and transplantation.

Results: In this retrospective cohort of 87 patients, 42 patients (48%) were treated with rhGH 3.9 years after the diagnosis at a median age of 7.4 years. Before the treatment start, they had a significant loss of height of –0.7 SDS (P<0.0001), 48% of them exhibiting growth failure < –2 SDS. Over 1.7 years of treatment, the median height gain was +0.7 SDS (P<0.0001). Growth outcome was negatively associated with an older age and end-stage renal disease at rhGH initiation. The 45 untreated patients during the same period experienced a loss of height of –0.6 DS (P=0.02) from diagnosis to transplantation besides a normal median height of +0.22 SDS and –0.42 SDS respectively. At transplantation, 26% of the treated and 9% of the untreated patients had growth failure < –2 SDS. RhGH therapy was initiated by a nephrologist in 52% cases and an endocrinologist in 48% cases. The initial prescription deviated from the marketing authorization criteria in 68% cases, mostly for 2 criteria (HtSDS > –2 SDS and age < 2 years). Median rhGH dosages were 0.044 and 0.036 mg/kg/day at initiation and during follow-up respectively.

Conclusion: RhGH therapy is effective in improving growth in CKD patients. However, the late initiation of rhGH therapy in patients with end-stage renal disease, the short duration of treatment and the insufficient rhGH dosage adjustment during follow-up might explain the lower gain of height observed in these patients than previously published.