An unusual ultrasound appearance of renal hemosiderosis in acute sickle cell nephropathy.

Sickle cell disease is the most common inherited blood disorder in the United States. The primary driver of pathology is microvascular occlusion which affects multiple organ systems including the kidney. The renal pathology usually manifests as hematuria, proteinuria, or microalbuminuria, and up to 10% of individuals with homozygous sickle cell disease (HbSS) develop renal failure over their lifetime.

Medical Student H&Ps: Do You Have to Observe Them All At Once?

Objectives: Direct observation of medical students' history and physical examination (H&P) skills by attendings is essential in ensuring trainees' competence. This study compared whether partial observations by multiple pediatric attendings across various clinical encounters versus a full observation by one attending affected students' performance on the pediatric Objective Structured Clinical Examination (OSCE) and the Year 3 Clinical Performance Examination (CPX3).

Methods: For the 2013-2014 and 2014-2015 academic years, 323 medical students submitted either H&P checklists completed by one attending observing an entire H&P (full observations) versus multiple attendings observing portions of the H&P (partial observations).

Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia-TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority trial.

Background: For children with sickle cell anaemia and high transcranial doppler (TCD) flow velocities, regular blood transfusions can effectively prevent primary stroke, but must be continued indefinitely. The efficacy of hydroxycarbamide (hydroxyurea) in this setting is unknown; we performed the TWiTCH trial to compare hydroxyurea with standard transfusions.

Methods: TWiTCH was a multicentre, phase 3, randomised, open-label, non-inferiority trial done at 26 paediatric hospitals and health centres in the USA and Canada.

Magnetic resonance imaging/angiography and transcranial Doppler velocities in sickle cell anemia: results from the SWiTCH trial.

The Stroke With Transfusions Changing to Hydroxyurea (SWiTCH) trial compared standard (transfusions/chelation) to alternative (hydroxyurea/phlebotomy) treatment to prevent recurrent stroke and manage iron overload in children chronically transfused over 7 years before enrollment. Standardized brain magnetic resonance imaging/magnetic resonance angiography (MRA) and transcranial Doppler (TCD) exams were performed at entry and exit, with a central blinded review. A novel MRA vasculopathy grading scale demonstrated frequent severe baseline left/right vessel stenosis (53%/41% ≥Grade 4); 31% had no vessel stenosis on either side.

Ameliorating attention problems in children with sickle cell disease: a pilot study of methylphenidate.

Objective: This pilot study examined whether methylphenidate (MPH) was effective in enhancing cognitive performance and attention for children with sickle cell disease (SCD) with cerebrovascular complications who evidence attention problems.

Methods: In this multisite, pilot study, we evaluated 2 separate double-blind controlled clinical trials, including a laboratory trial of the short-term efficacy of MPH, with the second study a 3-week home/school crossover trial evaluating the efficacy of MPH. The laboratory trial included 14 participants between the age of 7 and 16 years.

Effect of chronic transfusion therapy on progression of neurovascular pathology in pediatric patients with sickle cell anemia.

Background: Chronic blood transfusion (CBT) is currently the standard of care for primary and secondary stroke prevention in children with sickle cell anemia (SCA). However, the effect of CBT on cerebrovascular pathology is not well known.

Methods: We reviewed children with SCA receiving CBT for abnormal transcranial Doppler (TCD) [n=12] or cerebrovascular accident (CVA) [n=22].

Safety and efficacy of high dose intravenous desferrioxamine for reduction of iron overload in sickle cell disease.

Background: Patients with sickle cell disease (SCD) receiving chronic blood transfusions are at risk of developing iron overload and organ toxicity. Chelation therapy with either subcutaneous (SQ) desferrioxamine (DFO) or oral deferasirox is effective in preventing and reducing iron overload but poses significant challenges with patient compliance. Intravenous (IV) infusions of high dose DFO have been utilized in non-compliant patients with heavy iron overload in small case series.

Prevalence and clinical correlates of microalbuminuria in children with sickle cell disease.

Sickle cell disease (SCD) is associated with a large spectrum of renal abnormalities, one of which, microalbuminuria/proteinuria (MA/P), is a known predictor of end-stage renal disease. We studied 90 children with SCD (57% male; mean age 11.4 +/- 5.

Clinical events after surgical splenectomy in children with sickle cell anemia.

Purpose: Despite advances in immune prophylaxis, sepsis remains the most feared complication following splenectomy for acute splenic sequestration crisis (ASSC) in children with sickle cell anemia (SCA). We seek to investigate the true prevalence of sepsis and other complications of splenectomy in this patient population.

Methods: We reviewed the records of children with SCA (HbSS) who underwent splenectomy for ASSC between 1993 and 2008 at a single institution.

Outcome of splenectomy in children younger than 4 years with sickle cell disease.

Purpose: Removal of the spleen in patients younger than 4 years has been reported to carry an increased risk of postsplenectomy sepsis and has not been universally accepted. We reviewed our experience with splenectomy in children with acute splenic sequestration crisis (ASSC) younger than 4 years.

Methods: The study involved retrospective review of demographic and operative data, number of ASSC, operative complications, infections, and death.

Liver biopsy results in patients with sickle cell disease on chronic transfusions: poor correlation with ferritin levels.

Background: Chronic transfusions are effective in preventing stroke and other complications of sickle cell disease. The aim of this study was to determine whether serum ferritin levels correlated with liver iron content in sickle cell patients on chronic transfusion.

Procedure: Forty-four liver biopsy specimens from 38 patients with homozygous sickle cell anemia (HbSS) and one patient with sickle thalassemia receiving chronic transfusions were studied.

Radiographic predictors of neurocognitive functioning in pediatric Sickle Cell disease.

We compared magnetic resonance imaging (MRI), magnetic resonance angiography, and transcranial Doppler ultrasonography as predictors of specific neurocognitive functions in children with sickle cell disease. Participants were 27 children with sickle cell anemia (hemoglobin SS) who were participants in the Stroke Prevention Trial in Sickle Cell Anemia (STOP) and had no documented history of stroke. Children's MRIs were classified as normal or silent infarct, and their magnetic resonance angiograms were classified as normal or abnormal.

Transcranial Doppler ultrasonography and neurocognitive functioning in children with sickle cell disease.

Objective: We examined the relationship between cerebral blood flow velocity, measured by transcranial Doppler (TCD) ultrasonography, and neurocognitive functioning.

Methods: Participants were 60 children who had sickle cell disease (HbSS) and had no documented history of stroke. Children were classified according to Stroke Prevention Trial in Sickle Cell Anemia criteria (normal, conditional, and abnormal), and their performance was compared on measures of intellectual abilities, academic achievement, sustained attention/concentration, executive function, and parent and teacher ratings of executive function.