Barriers to and Facilitators of Iron Therapy in Children with Iron Deficiency Anemia.

Objective: To characterize barriers to and facilitators of successful iron therapy in young children with iron deficiency anemia (IDA) from an in-depth parental perspective.

Study Design: Prospective, mixed methods study of children age 9 months to 4 years with a diagnosis of nutritional IDA by clinical history and laboratory criteria and their parents. Clinical data were obtained from the electronic health record.

Factor VIII prophylaxis effects outweigh other hemostasis contributors in predicting severe haemophilia A joint outcomes.

Introduction: The Joint Outcome Study (JOS) demonstrated that previously untreated children with severe haemophilia A treated with prophylactic factor VIII (FVIII) concentrate had superior joint outcomes at age 6 years compared to those children treated episodically for bleeding. However, variation in joint outcome within each treatment arm was not well explained.

Aim: In this study, we sought to better understand variation in joint outcomes at age 6 years in participants of the JOS.

Disorders of Iron Metabolism: New Diagnostic and Treatment Approaches to Iron Deficiency.

Iron deficiency anemia is the leading cause of anemia worldwide and affects many young children and adolescent girls in the United States. Its signs and symptoms are subtle despite significant clinical effects. Iron deficiency anemia is diagnosed clinically by the presence of risk factors and microcytic anemia.

Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes.

Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder with isolated thrombocytopenia and hemorrhagic risk. While many children with ITP can be safely observed, treatments are often needed for various reasons, including to decrease bleeding, or to improve health related quality of life (HRQoL). There are a number of available second-line treatments, including rituximab, thrombopoietin-receptor agonists, oral immunosuppressive agents, and splenectomy, but data comparing treatment outcomes are lacking.

Physician decision making in selection of second-line treatments in immune thrombocytopenia in children.

Immune thrombocytopenia (ITP) is an acquired autoimmune bleeding disorder which presents with isolated thrombocytopenia and risk of hemorrhage. While most children with ITP promptly recover with or without drug therapy, ITP is persistent or chronic in others. When needed, how to select second-line therapies is not clear.

Effect of Low-Dose Ferrous Sulfate vs Iron Polysaccharide Complex on Hemoglobin Concentration in Young Children With Nutritional Iron-Deficiency Anemia: A Randomized Clinical Trial.

Importance: Iron-deficiency anemia (IDA) affects millions of persons worldwide, and is associated with impaired neurodevelopment in infants and children. Ferrous sulfate is the most commonly prescribed oral iron despite iron polysaccharide complex possibly being better tolerated.

Objective: To compare the effect of ferrous sulfate with iron polysaccharide complex on hemoglobin concentration in infants and children with nutritional IDA.

Iron Deficiency Anemia in Adolescents Who Present with Heavy Menstrual Bleeding.

Study Objective: To assess the clinical severity and initial treatment of iron deficiency anemia (IDA) in female adolescents with heavy menstrual bleeding (HMB) in our center.

Design: Retrospective cohort study of electronic medical records via search of administrative records using International Classification of Diseases Ninth Revision codes for IDA or unspecified anemia and disorders of menstruation.

Setting: Children's Medical Center in Dallas, Texas.

Intravenous Ferric Carboxymaltose in Children with Iron Deficiency Anemia Who Respond Poorly to Oral Iron.

Objective: To assess the benefits and risks of intravenous (IV) ferric carboxymaltose (FCM) in children with iron deficiency anemia (IDA).

Study Design: In a retrospective cohort study of patients seen at our center, we identified all FCM infusions in children with IDA over a 12-month period through a query of pharmacy records. Clinical data, including hematologic response and adverse effects, were extracted from the electronic medical record.

Deficiencies in the Management of Iron Deficiency Anemia During Childhood.

Limited high-quality evidence supports the management of iron deficiency anemia (IDA). To assess our institutional performance in this area, we retrospectively reviewed IDA treatment practices in 195 consecutive children referred to our center from 2006 to mid-2010. The majority of children were ≤4 years old (64%) and had nutritional IDA (74%).

Occult hemorrhage in children with severe ITP.

Little is known about the frequency and significance of clinically unapparent or occult hemorrhage in ITP. Therefore, we prospectively explored the sites and frequency of occult bleeding in children with severe ITP at diagnosis or upon symptomatic relapse in a prospective, single-institution cohort study of patients ≤ 18 years of age and a platelet count ≤ 10,000/mm(3) . Data collected included bleeding severity assessment, urinalysis, fecal occult blood testing, and non-contrast brain MRI.

Sports Participation in Children and Adolescents with Immune Thrombocytopenia (ITP).

We surveyed 278 pediatric hematologists/oncologists regarding how children with immune thrombocytopenia (ITP) are counseled for participation in sports. Results show substantial variation in physician perception of contact risk for different sports, and the advice offered about restriction of sport activities of affected children. Many physicians recommend restriction of sports when platelet counts are under 50 × 10(9) /L.

Management of iron deficiency anemia: a survey of pediatric hematology/oncology specialists.

Background: Iron deficiency anemia (IDA) is the most common hematologic condition in children and adolescents in the United States (US). No prior reports have described the management of IDA by a large cohort of pediatric hematology/oncology specialists.

Procedure: A 20-question electronic survey that solicited responses to two hypothetical cases of IDA was sent to active members of the American Society of Pediatric Hematology/Oncology (ASPHO) in the US.

Long-term use of the thrombopoietin-mimetic romiplostim in children with severe chronic immune thrombocytopenia (ITP).

Background: Treatment of chronic severe pediatric ITP is not well studied. In a phase 1/2 12-16-week study, 15/17 romiplostim-treated patients achieved platelet counts ≥50 × 10 /L, and romiplostim treatment was well tolerated. In a subsequent open-label extension (≤109 weeks), 20/22 patients received romiplostim; all achieved platelet counts >50 × 10 /L.

Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members.

Importance: Sickle cell disease (SCD) is a life-threatening genetic disorder affecting nearly 100,000 individuals in the United States and is associated with many acute and chronic complications requiring immediate medical attention. Two disease-modifying therapies, hydroxyurea and long-term blood transfusions, are available but underused.

Objective: To support and expand the number of health professionals able and willing to provide care for persons with SCD.

Immune thrombocytopenia and alopecia areata: spontaneous occurrence and resolution in the same patient.

Alopecia areata (AA) and immune thrombocytopenia (ITP) are autoimmune conditions occasionally encountered by pediatricians, but their simultaneous occurrence is rare. We describe here a 7-year-old female who acutely developed both AA and ITP. Within 3 months both conditions resolved spontaneously, suggesting a pathophysiologic relationship.

Diagnosis and management of iron deficiency anemia.

Iron deficiency anemia (IDA) is a common hematologic condition, affecting a substantial proportion of the world's women and young children. Optimal management of IDA requires an accurate diagnosis, identification and correction of the underlying cause, provision of medicinal iron therapy, and confirmation of treatment success. There are limited data to support current treatment approaches regarding oral iron preparation, dosing, monitoring, and duration of therapy.

"Packaging" of fetal hemoglobin in sickle cell anemia.

In this issue of Blood, Steinberg et al describe the clinical importance of the distribution or "packaging" of fetal hemoglobin (HbF) within erythrocytes of persons with sickle cell anemia.

Burden of diagnostic radiation exposure in children with sickle cell disease.

Children with sickle cell disease (SCD) are repeatedly exposed to diagnostic radiation. We identified 938 children with SCD who had 9,246 radiographic tests. Mean number of tests/patient was 9.

Blood pressure abnormalities in children with sickle cell anemia.

Background: Kidney disease is an important cause of morbidity and mortality in patients with sickle cell anemia (SCA). The factors that affect progression of renal disease are unknown, especially in children and adolescents. Alterations in blood pressure, including hypertension and lack of the normal nocturnal dip in blood pressure, are important determinants of diabetic nephropathy and other renal diseases and may play a role in sickle cell nephropathy.