RESUMEN
BACKGROUND: Stem cell transplantation (SCT) outcomes have improved over the last three decades, with many patients being rescued with this treatment. However, improved outcomes have led to issues with long-term sequelae. One of these sequelae in children is renal dysfunction, an index of which is estimated using glomerular filtration rate (eGFR). PROCEDURE: We retrospectively analyzed eGFR in 83 pediatric patients who received SCT. Data from all patients extended up to 12 months or more post SCT. The median follow-up time was 127.7 months (range 12.0-268.8 months). RESULTS: Eighteen patients (21.7%) had low eGFR (<90 ml/min/1.73 m2 ) post SCT. Cumulative incidence of low eGFR was 25.8 ± 2.0%. Nine (10.6%) patients had a low eGFR pre-SCT. However, pre- and post-SCT incidence of low eGFR were not correlated. Meanwhile, only two patients (2.4%) exhibited severe renal dysfunction, with eGFRs < 60 ml/min/1.73 m2 . Independent risk factors for low eGFR were solid tumor and use of fludarabine. Moreover, age at SCT ≥ 7 years was also a long-term post-SCT risk factor for low eGFR in all patients. CONCLUSION: Independent post-SCT long-term risk factors for low eGFR in children were solid tumor and use of fludarabine. Moreover, age at SCT ≥ 7 years was a post-SCT long-term risk factor for low eGFR across all patients.
Asunto(s)
Tasa de Filtración Glomerular , Trasplante de Células Madre Hematopoyéticas/efectos adversos , Enfermedades Renales/epidemiología , Neoplasias/tratamiento farmacológico , Adolescente , Adulto , Niño , Preescolar , Femenino , Humanos , Incidencia , Lactante , Recién Nacido , Enfermedades Renales/etiología , Masculino , Estudios Retrospectivos , Adulto JovenRESUMEN
Anti-muscle-specific tyrosine kinase antibody (MuSK-Ab) is the second most frequent autoantibody identified in adult patients with myasthenia gravis (MG). Adult patients with MuSK-Ab demonstrate characteristic clinical features but very little information is available for childhood-onset patients with MuSK-positive MG. We report a childhood-onset female patient with MuSK-positive MG. This patient showed basic clinical features compatible with adult-onset MuSK-positive MG, but some features, including spontaneous improvement, are distinct from those in adult patients. Serial examination of MuSK-Ab titers revealed a gross correlation with clinical severity despite significantly high titers throughout the clinical course. Therefore, childhood-onset MuSK-positive MG may demonstrate a distinct clinical characteristics in the early period of illness.