ABSTRACT
Background: The purpose of this study is to evaluate the effectiveness and safety of switching from immediate-release (IR) to extended-release (ER) cysteamine in patients with nephropathic cystinosis (NC) in Spain. Methods: We conducted an observational, retrospective, multicentre study in NC patients who received IR cysteamine for at least 12 months, switched to ER cysteamine, and received it for at least 6 months before inclusion. Results: Data were collected from nine patients (four children, five adults) 36 months before and after the switch. Despite the highly selected population, an improvement in growth, particularly in children and a significant reduction in hospitalization days was observed. A decrease in halitosis, body odour and gastrointestinal effects was reported in most of the patients who suffered before the switch, and the use of proton pump inhibitors (PPIs) decreased in some patients. The estimated glomerular filtration rate (eGFR) remained stable in patients with preserved kidney function. No significant changes in white blood cell (WBC) cystine levels were observed after the switch. There was no significant difference in the cysteamine dose received. However, some patients were receiving <50% of the recommended dose of cysteamine before and after the switch and showed elevated levels of WBC cystine. Conclusions: Switching from IR to ER cysteamine in clinical practice reduces hospital stays, improves nutritional status and growth in paediatric patients and could help to enhance treatment tolerability by reducing side effects. Furthermore, the dosing of ER cysteamine could promote therapeutic compliance and positively affect the quality of life of the NC population.
ABSTRACT
AIM: To describe a series of patients who received intravenous immunoglobulin (IVIg) for the treatment of neonatal hyperbilirubinaemia and developed necrotizing enterocolitis (NEC) shortly thereafter. POPULATION AND RESULTS: We describe three healthy breastfed newly born infants with isoimmunization-derived hyperbilirubinaemia refractory to phototherapy who were treated with IVIg. Shortly after the perfusion finished they developed clinical and radiological signs compatible with NEC and needed antibiotic therapy, prolonged parenteral nutrition and even surgery in one case. Other conditions such as septicaemia or coagulopathy were ruled out. Microscopic examination of the resected intestine revealed the presence of disseminated thrombi obstructing multiple minor vessels of the mesenteric circulation. CONCLUSION: IVIg in the newborn period should be cautiously employed and always administered under strict medical control.
