RESUMO
Severe aplastic anemia (SAA) is a rare potentially fatal hematologic disorder. Although overall outcomes with treatment are excellent, there are variations in management approach, including differences in treatment between adult and pediatric patients. Certain aspects of treatment are under active investigation in clinical trials. Because of the rarity of the disease, some pediatric hematologists may have relatively limited experience with the complex management of SAA. The following recommendations reflect an up-to-date evidence-based approach to the treatment of children with relapsed or refractory SAA.
RESUMO
Severe aplastic anemia (SAA) is a rare potentially fatal hematologic disorder. Although overall outcomes with treatment are excellent, there are variations in management approach, including differences in treatment between adult and pediatric patients. Certain aspects of treatment are under active investigation in clinical trials. Because of the rarity of the disease, some pediatric hematologists may have relatively limited experience with the complex management of SAA. The following recommendations reflect an up-to-date evidence-based approach to the treatment of children with newly diagnosed SAA.
RESUMO
Daratumumab, a CD38 monoclonal antibody that has been FDA-approved to treat multiple myeloma, has acquired popularity and is used off-label for both auto- and alloantibody mediated disorders, particularly in refractory/resistant circumstances. Much of the published data for its use in pediatric blood disorders has been in post-transplant autoimmune cytopenias. Here we describe three patients in whom daratumumab was used outside of post-transplant autoimmune cytopenias, highlighting further potential uses of this medication.
Assuntos
Anemia Hemolítica Autoimune , Mieloma Múltiplo , Humanos , Criança , Anticorpos Monoclonais/efeitos adversos , Anemia Hemolítica Autoimune/tratamento farmacológico , Anemia Hemolítica Autoimune/etiologiaRESUMO
Wiskott-Aldrich syndrome (WAS) is a rare X-linked recessive inborn error of immunity (IEI) first described in 1937. Classic WAS is characterized by the triad of thrombocytopenia with small platelets, recurrent infections due to combined immunodeficiency, and eczema. Hematopoietic stem cell transplantation (HSCT) was the only curative option available for five decades, with excellent outcomes reported for matched sibling donors (MSD) and matched unrelated donors (MUD). More recently, alternative donor transplants such as umbilical cord blood (UCB) and haploidentical transplant have emerged as viable options due to improvements in better graft selection, cell dosing, and effective allograft manipulation measures. Gene therapy is another potential curative option with promising results, yet currently is offered only as part of a clinical trial.
RESUMO
BACKGROUND: Hematopoietic stem cell transplant (HSCT) can cure or alleviate a wide range of nonmalignant childhood conditions. However, few studies have examined longitudinal national trends of frequency or short-term complications of HSCT before 2006 when an HSCT became a reportable procedure by US law. By using a US nationally representative database, we conducted nationwide longitudinal analyses on demographics, in-hospital mortality, and short-term complications in nonmalignant HSCT from 2000 to 2012. PROCEDURE: We analyzed 2504 admissions for children < 20 years old who underwent an allogeneic HSCT for a nonmalignant condition by using the Kids' Inpatient Database for the years 2000, 2003, 2006, 2009, and 2012. Changes in in-hospital mortality and other outcomes were assessed over the study period using weighted analyses, which enabled generation of national estimates in each year. RESULTS: The number of admissions for HSCT increased from 334 to 667 from 2000 to 2012, respectively; among them, the use of bone marrow decreased (66.5% to 34.1%, P < 0.001). In-hospital mortality declined (13.4% to 7.1%, P = 0.04), as did bacteremia (28.7% to 10.1%, P < 0.001) and vascular catheter infections (18.8% to 8.7%, P = 0.006), but cytomegalovirus infections increased (4.9% to 15.9%, P < 0.001), as did adenovirus infections (1.8% to 6.9%, P < 0.001) from 2000 to 2012. CONCLUSION: Population-based analyses demonstrated a substantial expansion of the utilization of HSCT occurred for pediatric nonmalignancies from 2000 to 2012 in the United States, whereas the in-hospital mortality declined by approximately a half. Further research is needed to identify distinct contributing factors.
Assuntos
Bases de Dados Factuais , Doenças Hematológicas/mortalidade , Transplante de Células-Tronco Hematopoéticas/mortalidade , Mortalidade Hospitalar/tendências , Hospitalização/estatística & dados numéricos , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , Seguimentos , Doenças Hematológicas/patologia , Doenças Hematológicas/terapia , Humanos , Lactente , Recém-Nascido , Estudos Longitudinais , Masculino , Prognóstico , Estudos Retrospectivos , Fatores de Risco , Taxa de Sobrevida , Transplante Autólogo , Adulto JovemRESUMO
Massive splenic infarction (MSI) is a rare complication of sickle cell disease, as the spleen generally atrophies within the first few years of life. We report a case of MSI in a 12-year-old boy with homozygous sickle cell anemia (Hb SS) whose chronic transfusion therapy resulted in hypersplenism. The occurrence of a complicated MSI in our patient should perhaps further encourage elective splenectomy in such patients, despite known potential perioperative complications and postsplenectomy risks of infection and thrombosis.