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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. Results Of 1,217 recipients, 398 (32.7%) reported regularly treating IDA and completed the survey. In a toddler with nutritional IDA, 15% (N = 61) of respondents reported ordering no diagnostic test beyond a complete blood count. Otherwise, wide variability in laboratory testing was reported. For treatment, most respondents would prescribe ferrous sulfate (N = 335, 84%) dosed at 6 mg/kg/day (N = 248, 62%) divided twice daily (N = 272, 68%). The recommended duration of iron treatment after resolution of anemia and normalized serum ferritin varied widely from 0 to 3 months. For an adolescent with heavy menstrual bleeding and IDA, most respondents recommended ferrous sulfate (N = 327, 83%), with dosing based on the number of tablets daily. For IDA refractory to oral treatment, intravenous iron therapy was recommended most frequently, 48% (N = 188) using iron sucrose, 17% (N = 68) ferric gluconate, and 15% (N = 60) low molecular weight iron dextran. Conclusion The approach to diagnosis and treatment of IDA in childhood was widely variable among responding ASPHO members. Given the lack of an evidence base to guide clinical decision making, further research investigating IDA management is needed. Pediatr Blood Cancer 2015;62:842–846. © 2015 Wiley Periodicals, Inc.

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%). In 11- to 18-year-old patients (31%), the primary etiology was menorrhagia (42%). Many were referred directly to the emergency department and/or prescribed iron doses outside the recommended range. Poor medication adherence and being lost-to-follow-up were common. Substantial improvements are required in the management of IDA.

Background Existing therapies for recurrent or refractory histiocytoses, including Langerhans cell histiocytosis (LCH), juvenile xanthogranuloma (JXG), and Rosai-Dorfman disease (RDD), have limited effectiveness. We report our experience with using clofarabine as therapy in children with recurrent or refractory histiocytic disorders, including LCH (11 patients), systemic JXG (4 patients), and RDD (3 patients).

Background Sickle cell disease (SCD) is an inherited blood disorder characterized by a chronic hemolytic anemia that can contribute to fatigue and global cognitive impairment in patients. The study objective was to report on the feasibility, reliability, and validity of the PedsQL™ Multidimensional Fatigue Scale in SCD for pediatric patient self-report ages 5–18 years and parent proxy-report for ages 2–18 years.

Background Iron deficiency anemia (IDA) in children is usually treated with oral iron, yet many respond poorly. Intravenous low molecular weight iron dextran (LMWID) offers the opportunity of employing a single outpatient infusion to correct the anemia and reduce the overall burden of treatment, but its use in children has been limited due to concerns of serious adverse effects. In this study we report our initial experience using LMWID in children with iron deficiency in whom oral iron was ineffective. Methods We performed a case series of LMWID treatment of children with IDA of diverse etiologies who were poorly responsive to oral iron therapy with the aim of measuring its efficacy and adverse effects. LMWID was administered as a total dose infusion over 60 minutes in the outpatient setting. Results Thirty-one patients age 11 months to 18 years received intravenous LMWID, and 24 were evaluable for hematologic response. Median hemoglobin increments were respectively 3.5, 1.9, and 1.8 g/dl in patients with IDA due to poor nutrition (n = 11), chronic blood loss (n = 13), and miscellaneous causes (n = 7). Two thirds of evaluable patients had a complete hematologic response. Nine of the patients (29%) had mild non-specific adverse effects upon initiation of the LMWID infusion. Conclusions LMWID as a total dose infusion was well tolerated and effective in a heterogeneous group of children and adolescents with IDA who were refractory to oral iron therapy. Transient reactions were common but not serious. Pediatr Blood Cancer 2013;60:1747–1752. © 2013 Wiley Periodicals, Inc.

Background Sickle cell disease (SCD) is an inherited chronic disease that is characterized by complications such as recurrent painful vaso-occlusive events that require frequent hospitalizations and contribute to early mortality. The objective of the study was to report on the initial measurement properties of the new PedsQL™ SCD Module for pediatric patient self-report ages 5–18 years and parent proxy-report for ages 2–18 years. Procedure The 43-item PedsQL™ SCD Module was completed in a multisite study by 243 pediatric patients with SCD and 313 parents. Participants also completed the PedsQL™ 4.0 Generic Core Scales and PedsQL™ Multidimensional Fatigue Scale. Results The PedsQL™ SCD Module Scales evidenced excellent feasibility, excellent reliability for the Total Scale Scores (patient self-report α = 0.95; parent proxy-report α = 0.97), and good reliability for the nine individual scales (patient self-report α = 0.69–0.90; parent proxy-report α = 0.83–0.97). Intercorrelations with the PedsQL™ Generic Core Scales and PedsQL™ Multidimensional Fatigue Scales were medium (0.30) to large (0.50) range, supporting construct validity. PedsQL™ SCD Module Scale Scores were generally worse for patients with severe versus mild disease. Confirmatory factor analysis demonstrated an acceptable to excellent model fit. Conclusions The PedsQL™ SCD Module demonstrated acceptable measurement properties. The PedsQL™ SCD Module may be utilized in the evaluation of SCD-specific health-related quality of life in clinical research and practice. In conjunction with the PedsQL™ Generic Core Scales and the PedsQL™ Multidimensional Fatigue Scale, the PedsQL™ SCD Module will facilitate the understanding of the health and well-being of children with SCD. Pediatr Blood Cancer 2013;60:1338–1344. © 2013 Wiley Periodicals, Inc.

published reports describe quality improvement initiatives designed to decrease TTA in pediatric patients with FN. Underly- ing these reports is the unstated assumption that decreased TTA is associated with improved outcomes of pediatric patients with FN (16-18). Therefore, we sought to test this assumption and deter- mine whether TTA is associated with outcomes of FN in the setting of pediatric cancer. We hypothesized that prolonged TTA would be associated with poor outcomes in FN. Background. Time-to-antibiotic (TTA) administration is a widely used quality-of-care measure for children with cancer and febrile neutropenia (FN). We sought to determine whether TTA is associat- ed with outcomes of FN. Procedure. A single-center, retrospective cohort study was conducted of 1,628 FN admissions from 653 patients from 2001 to 2009. Outcome variables included (1) an adverse event (AE) composite of in-hospital mortality, pediatric intensive care unit (PICU) admission within 24 hours of presenta- tion, and/or fluid resuscitation � 40 ml/kg within 24 hours of pre- sentation and (2) length of stay (LOS). TTA was measured as a continuous variable and in 60-minute intervals. Mixed regression models were constructed to evaluate associations of TTA with the outcome variables after adjusting for relevant covariates including cancer diagnosis, degree of myelosuppression, and presence of bacteremia. Results. The composite AE outcome occurred in 11.1% of admissions including 0.7% in-hospital mortality, 4.7% PICU admission, and 10.1% fluid resuscitation. In univariate analy- sis, TTA was associated with the composite AE outcome (Odds Ratio (OR) 1.29, 95% CI 1.02-1.64) but not LOS. In multivariate analysis, after adjustment for relevant covariates, 60-minute TTA intervals were associated with the composite AE outcome (61- 120 minutes vs. � 60 minutes, OR 1.81, 95% CI 1.01-3.26). Unex- pectedly, admission from the emergency department (ED) was also independently associated with the composite AE outcome (ED vs. clinic, OR 3.15, 95% CI 1.95-5.09). Conclusions. TTA and presen- tation to the ED are independently associated with poor outcomes of FN. Pediatr Blood Cancer 2013;60:1299-1306. 2013 Wiley Periodicals, Inc.

Background The success of primary stroke prevention for children with sickle cell disease (SCD) throughout the United States is unknown. Therefore, we aimed to generate national incidence rates of hospitalization for stroke in children with sickle cell disease (SCD) before and after publication of the Stroke Prevention Trial in Sickle Cell Anemia (STOP trial) in 1998.

Objective To estimate national hospitalization rates for invasive pneumococcal disease (IPD) in children with sickle cell disease (SCD) before and after the 2000 licensure of the heptavalent pneumococcal conjugate vaccine (PCV7). Procedure We performed a retrospective trend analysis of the 1994–2007 Nationwide Inpatient Sample databases. Hospitalizations involving children with SCD and IPD were identified by ICD-9CM code. The primary outcomes, the annual hospitalization rate for IPD in children with SCD and the proportion of hospitalizations for IPD per 100 total SCD hospitalizations, were analyzed using multivariable linear regression and contingency analysis, respectively. Results A total of 1,242 hospitalizations for IPD in SCD patients were identified from 1994–2007, with a mortality rate of 2.4%. The national mean annual rate of IPD hospitalization decreased by 65%, from 131.8 cases/year from 1994 to 2000 to 45.5 cases/year from 2001 to 2007 (P = 0.001). The national proportion of hospitalizations for IPD per 100 total SCD hospitalizations decreased from 0.4 to 0.15 (P

Sickle cell disease (SCD) affects 70,000–100,000 people in the United States, accounting for nearly 100,000 hospitalizations annually [1]. In 2004, the average cost per SCD hospitalization was $6,223, with an estimated aggregate cost of $488 million [1]. Despite the large number and cost of hospitalizations in SCD patients, the quality of care (QOC) for these hospitalizations has not been studied sufficiently. In other medical conditions, frequently studied factors associated with QOC-related outcomes are hospital volume, hospital teaching status, patient socioeconomic status (SES), and patient insurance coverage. Most published reports have demonstrated that higher hospital volumes are associated with improved QOC, [2] an association first established in the surgical literature [3]. More recently, the association of hospital volume and outcomes has been reported for several medical conditions, including acute myocardial infarction (AMI) [4] and HIV/AIDS [5]. Similarly, lower SES and lack of health insurance have been consistently associated with poorer QOC [6–9]. The only published report investigating SES in SCD found no association between indirect SES markers and QOC-related outcomes in children [10]. The association between teaching status and QOC has been inconsistent [11]. To our knowledge, no prior study has investigated the effects of multiple potential determinants of QOC on QOC-related outcomes in SCD. We hypothesized that higher hospital volumes, specifically greater volume of SCD hospitalizations, are associated with improved QOC outcomes.

Time-to-antibiotic administration (TTA) has been suggested as a quality-of-care (QOC) measure for pediatric oncology patients with febrile neutropenia (FN). Unknown, however, is to what extent pediatric oncology centers utilize TTA. Therefore, we designed and administered an electronic survey (68% response rate) of programs in the Children's Oncology Group to assess TTA utilization. Nearly half of respondents track TTA. Most reported using a benchmark of less than 60 minutes from arrival. TTA is a commonly used QOC measure for pediatric FN despite an absence of studies establishing its validity and a lack of data supporting its impact on outcomes of FN.

Patients with sickle cell disease (SCD) have an increased risk of invasive bacterial infection because of hyposplenism. Bordetella holmesii is a recently described Gram-negative coccobacillus with an apparent predilection for asplenic hosts. We report two patients with SCD and B. holmesii bacteremia. Fastidious growth in culture and a typically uncomplicated clinical course distinguish B. holmesii infection from other invasive bacterial infections in SCD. Providers for patients with SCD should be aware of this pathogen and ensure that their microbiology laboratories are capable of isolating and identifying this organism.