RESUMO
OBJECTIVES: The aim of the study was to assess the treatment efficacy of transanal irrigation and parental satisfaction in children with intractable functional constipation (FC) treated with Peristeen. METHODS: Cross-sectional survey study among parents of children (age 0-18 years) treated with Peristeen for FC (based on the Rome III criteria). Anonymous questionnaires were sent out to parents via mail, these consisted of 25 self-developed, multiple-choice questions regarding the use of Peristeen, current gastrointestinal symptoms, adverse effects of Peristeen, concomitant medication use, and parental satisfaction. RESULTS: Out of 91 invited families, 67 (74%) returned the questionnaire. In total, 84% of patients experienced fecal incontinence prior to treatment. Out of all children who still used Peristeen at the time of survey (nâ=â49), fecal incontinence had resolved completely in 41%, 12% experienced occasional episodes of fecal incontinence (<1 episode per week) and the remaining 47% still experienced episodes of fecal incontinence regularly (≥1 time per week). A total of 28 children (42%) experienced pain during rectal irrigation, especially during insertion of the catheter, inflating the balloon, or during irrigation. Overall, 86% of the parents were satisfied with the result of transanal irrigation and 67% reported that they would continue using transanal irrigation for the treatment of their child's symptoms. CONCLUSIONS: Transanal irrigation may be effective in the treatment of children with FC and renders a high parental satisfaction. Future prospective studies, preferably RCTs, are necessary to further evaluate this treatment option.
Assuntos
Constipação Intestinal/terapia , Irrigação Terapêutica/métodos , Adolescente , Canal Anal , Criança , Pré-Escolar , Estudos Transversais , Feminino , Pesquisas sobre Atenção à Saúde , Humanos , Lactente , Recém-Nascido , Masculino , Pais , Satisfação do Paciente , Irrigação Terapêutica/instrumentação , Resultado do TratamentoRESUMO
Deficiency of the phospholipid flippase ATPase, aminophospholipid transporter, class I, type 8B, member 1 (ATP8B1) causes progressive familial intrahepatic cholestasis type 1 (PFIC1) and benign recurrent intrahepatic cholestasis type 1 (BRIC1). Apart from cholestasis, many patients also suffer from diarrhea of yet unknown etiology. Here we have studied the hypothesis that intestinal ATP8B1 deficiency results in bile salt malabsorption as a possible cause of PFIC1/BRIC1 diarrhea. Bile salt transport was studied in ATP8B1-depleted intestinal Caco-2 cells. Apical membrane localization was studied by a biotinylation approach. Fecal bile salt and electrolyte contents were analyzed in stool samples of PFIC1 patients, of whom some had undergone biliary diversion or liver transplantation. Bile salt uptake by the apical sodium-dependent bile salt transporter solute carrier family 10 (sodium/bile acid cotransporter), member 2 (SLC10A2) was strongly impaired in ATP8B1-depleted Caco-2 cells. The reduced SLC10A2 activity coincided with strongly reduced apical membrane localization, which was caused by impaired apical membrane insertion of SLC10A2. Moreover, we show that endogenous ATP8B1 exists in a functional heterodimer with transmembrane protein 30A (CDC50A) in Caco-2 cells. Analyses of stool samples of post-transplant PFIC1 patients demonstrated that bile salt content was not changed, whereas sodium and chloride concentrations were elevated and potassium levels were decreased. The ATP8B1-CDC50A heterodimer is essential for the apical localization of SLC10A2 in Caco-2 cells. Diarrhea in PFIC1/BRIC1 patients has a secretory origin to which SLC10A2 deficiency may contribute. This results in elevated luminal bile salt concentrations and consequent enhanced electrolyte secretion and/or reduced electrolyte resorption.
Assuntos
Adenosina Trifosfatases/metabolismo , Proteínas de Membrana/metabolismo , Transportadores de Ânions Orgânicos Dependentes de Sódio/metabolismo , Simportadores/metabolismo , Adenosina Trifosfatases/química , Adenosina Trifosfatases/genética , Ácidos e Sais Biliares/metabolismo , Transporte Biológico/genética , Western Blotting , Células CACO-2 , Membrana Celular/metabolismo , Colestase Intra-Hepática/genética , Colestase Intra-Hepática/metabolismo , Fezes/química , Humanos , Mucosa Intestinal/metabolismo , Proteínas de Membrana/química , Proteínas de Membrana/genética , Mutação , Multimerização Proteica , Interferência de RNA , Reação em Cadeia da Polimerase Via Transcriptase Reversa , Ácido Taurocólico/metabolismo , Ácido Taurocólico/farmacocinéticaRESUMO
Posaconazole (PSZ) may be an attractive alternative for antifungal prophylaxis in children with chronic granulomatous disease. Experience with PSZ in pediatric patients is limited, and no specific dose recommendations exist. A twice daily dosing algorithm based on allometric scaling (body-weight based) for PSZ results in adequate exposure and appears to be safe in children with chronic granulomatous disease.
