RESUMEN
An eight-yr-old female with a history of multifocal lymphangioendotheliomatosis and thrombocytopenia presented for MVT. The patient had multiple vascular lesions in the skin and stomach in infancy. Although her cutaneous lesions resolved with vincristine and methylprednisolone, her gastric lesions persisted. Eight yr later, she was diagnosed with portal hypertension and decompensating liver function despite therapy with bevacizumab, propranolol, furosemide, and spironolactone. Upon presentation, she was found to have a Kasabach-Merritt-like coagulopathy in association with multiple lesions in her GI tract and persistent gastric lesions. Although treatment with methylprednisolone and sirolimus normalized her coagulation factors and d-dimer levels, she never developed sustained improvement in her thrombocytopenia. Her liver function continued to deteriorate and she developed hepatorenal syndrome. Given better outcomes after OLT in comparison with MVT, she underwent OLT, with the plan to manage her GI lesions with APC post-transplant. Post-transplant, her liver function and coagulopathy normalized, and GI tract lesions disappeared upon screening with capsule endoscopy. The patient is doing well, without recurrence of either GI lesions or thrombocytopenia, at 18 months after transplantation.
Asunto(s)
Trasplante de Hígado/métodos , Linfangioma/complicaciones , Linfangioma/cirugía , Trombocitopenia/complicaciones , Trombocitopenia/cirugía , Coagulación Sanguínea , Factores de Coagulación Sanguínea , Niño , Colestasis , Femenino , Productos de Degradación de Fibrina-Fibrinógeno/química , Tracto Gastrointestinal/patología , Síndrome Hepatorrenal/complicaciones , Humanos , Hipertensión Portal , Metilprednisolona/administración & dosificación , Estómago/patología , Resultado del Tratamiento , Vincristina/administración & dosificaciónRESUMEN
OBJECTIVES: ImproveCareNow (ICN) is the largest pediatric learning health system in the nation and started as a quality improvement collaborative. To test the feasibility and validity of using ICN data for clinical research, we evaluated the effectiveness of anti-tumor necrosis factor-α (anti-TNFα) agents in the management of pediatric Crohn disease (CD). METHODS: Data were collected in 35 pediatric gastroenterology practices (April 2007 to March 2012) and analyzed as a sequence of nonrandomized trials. Patients who had moderate to severe CD were classified as initiators or non-initiators of anti-TNFα therapy. Among 4130 patients who had pediatric CD, 603 were new users and 1211 were receiving anti-TNFα therapy on entry into ICN. RESULTS: During a 26-week follow-up period, rate ratios obtained from Cox proportional hazards models, adjusting for patient and disease characteristics and concurrent medications, were 1.53 (95% confidence interval [CI], 1.20-1.96) for clinical remission and 1.74 (95% CI, 1.33-2.29) for corticosteroid-free remission. The rate ratio for corticosteroid-free remission was comparable to the estimate produced by the adult SONIC study, which was a randomized controlled trial on the efficacy of anti-TNFα therapy. The number needed to treat was 5.2 (95% CI, 3.4-11.1) for clinical remission and 5.0 (95% CI, 3.4-10.0) for corticosteroid-free remission. CONCLUSIONS: In routine pediatric gastroenterology practice settings, anti-TNFα therapy was effective at achieving clinical and corticosteroid-free remission for patients who had Crohn disease. Using data from the ICN learning health system for the purpose of observational research is feasible and produces valuable new knowledge.
Asunto(s)
Enfermedad de Crohn/tratamiento farmacológico , Factor de Necrosis Tumoral alfa/antagonistas & inhibidores , Adolescente , Investigación Biomédica , Ensayos Clínicos Controlados como Asunto , Estudios de Factibilidad , Femenino , Humanos , Masculino , PediatríaRESUMEN
UNLABELLED: There is significant variation in diagnostic testing and treatment for inflammatory bowel disease. Quality improvement science methods can help address unwarranted variations in care and outcomes. METHODS: The ImproveCareNow Network was established under the sponsorship of the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition and the American Board of Pediatrics as a prototype for a model of improving subspecialty care that included three components: 1) creating enduring multicenter collaborative networks of pediatric subspecialists, 2) sharing of performance data collected in patient registries, and 3) training in quality improvement. The network began with a focus on improving initial diagnostic testing and evaluation, the classification of the severity and extent of disease, the detection and treatment of inadequate nutrition and growth, and the appropriate dosing of immunomodulator medications. Changes are based on an evidence-based model of chronic illness care involving the use of patient registries for population management, previsit planning, decision support, promoting self-management, and auditing of care processes. RESULTS: Currently, patients are being enrolled at 23 sites. Through 2009, data have been analyzed on over 2500 patients from over 7500 visits. Initial results suggest improvements in both care processes (e.g., appropriate medication dosing and completion of a classification bundle that includes the patient's diagnosis, disease activity, distribution and phenotype, growth status, and nutrition status) and outcomes (e.g., the percentage of patients in remission). CONCLUSIONS: These improvements suggest that practice sites are learning how to apply quality improvement methods to improve the care of patients.
Asunto(s)
Enfermedades Inflamatorias del Intestino/terapia , Pediatría/normas , Mejoramiento de la Calidad , Calidad de la Atención de Salud , Niño , HumanosRESUMEN
Allogeneic hematopoietic stem cell transplantation has been shown to correct or improve a variety of autoimmune disorders. This has not been reported for celiac disease, but transmission to a hematopoietic stem cell transplantation recipient from a donor with celiac disease has been reported. We report a 12-year-old girl with celiac disease who was diagnosed with acute leukemia and received an allogeneic hematopoietic stem cell transplant. Her celiac disease resolved after the hematopoietic stem cell transplant.
Asunto(s)
Enfermedad Celíaca/terapia , Trasplante de Células Madre Hematopoyéticas , Leucemia Mieloide Aguda/terapia , Enfermedad Celíaca/complicaciones , Niño , Femenino , Humanos , Leucemia Mieloide Aguda/complicaciones , Hermanos , Trasplante HomólogoRESUMEN
Severe gastrointestinal bleeding in the newborn period is a serious but uncommon phenomenon that has a broad differential diagnosis. In the following case report we describe a rare phenomenon in which a newborn presents with severe hematemesis, hematochezia, and thrombocytopenia that are resistant to repeated platelet and packed red blood cell transfusions. Previous cases have been reported, but none of the patients described presented within the first 8 days of life. The early age of presentation and refractory nature of this disease entity to multiple therapies make it a diagnostic and therapeutic dilemma for all physicians involved in the care of newborns.