Caring for a child with sickle cell disease poses extra demands on parents, both practically and psychologically, which may influence their quality of life. Since families of children with sickle cell disease in the Netherlands usually belong to immigrant communities with a low socio-economic status, there may be an additional strain on caregivers. The aim of the present study was to evaluate the quality of life of caregivers of children with sickle cell disease. The quality of life of female caregivers of sickle cell disease patients, measured with the TNO-AZL Adult Quality of Life questionnaire, was compared to the norm data of healthy Dutch females (n=700) and female caregivers of healthy children with the same socio-economic status and ethnic background (socio-economic status control group). Groups were compared by the Mann-Whitney U test. Point estimates and 95% confidence intervals of the median difference are presented. The results of questionnaires of 54 caregivers of children with sickle cell disease and 28 caregivers of a control group of the same socio-economic status were analyzed. Caregivers of patients with sickle cell disease had a significantly lower quality of life on all subscales compared to the Dutch norm population. Compared to the control group of the same socio-economic status, the quality of life of caregivers of patients with sickle cell disease was significantly lower on the subscales depressive moods, daily activities and vitality. In this first study reporting on the quality of life of caregivers of children with sickle cell disease, we demonstrate a reduced quality of life in these caregivers compared to the healthy Dutch female population and caregivers of healthy children with the same socio-economic status.Key words: sickle cell disease, quality of life, caregivers. Citation

There is no instrument to measure severity of sickle cell disease (SCD) in pediatric patients that is generally accepted. The aim of this study was to develop and validate a severity index for SCD in children. We developed an index consisting of 12 items and tested its validity of the index using data from 92 children. We tested whether different scores were obtained for patients classified by severity both subjectively and objectively by a partially validated existing index. Furthermore, we tested whether the index could differentiate patients classified according to genotype or the number of a-gene deletions and evaluated whether the score on the index was correlated with the average number and days of hospitalizations/year, age and a risk of death score. We explored the effect of three different weighting systems (Score A, B, and C) to summarize these items. All weightings demonstrated a significant difference between the scores of mild, moderate, and severely affected patients, as classified by a subjective rating or with an existing index (P < 0.01). The index clearly differentiated patients by genotype (P < 0.01) or a-gene deletions (P < 0.01). The correlation with hospitalization was moderate. Age and the risk of death score were weakly associated with the pediatric severity index for SCD. This is the first pediatric SCD severity index that was developed and validated using modern clinimetric methodology. The validity and reliability of this index should be further evaluated in a prospective study including a larger cohort, preferably diagnosed at birth. Am. J. Hematol. 85:746-751, 2010. V

SUMMARY:Arterial spin-labeling (ASL) is a relatively new and noninvasive MR imaging technique, used to measure cerebral blood flow (CBF). Scanning time and reproducibility remain important issues in the clinical applicability of ASL. We expected both to benefit from higher field strengths. We describe that when performing ASL at 3T, 20 averages suffice to obtain steady and reproducible CBF values. Scanning time can be as short as 3 minutes.A rterial spin labeling (ASL) is a noninvasive MR imaging technique, used for visualization and quantification of cerebral perfusion. Cerebral blood flow (CBF) values measured by ASL are comparable with CBF values measured by conventional techniques (eg, positron-emission tomography or susceptibility-weighted MR imaging). ASL is based on magnetic labeling of arterial blood water protons, which are used as an endogenous tracer of flow. Magnetic inversion takes place in a plane proximal to the brain. The decay rate of the labeled spins is sufficiently long to visualize perfusion of brain vasculature and microvasculature. Perfusion images are obtained by subtraction of successively acquired labeled and nonlabeled control images. In general, 40 to 60 paired acquisitions are averaged to improve perfusion signal intensity. ASL sequences differ in the way magnetic labeling is applied and are commonly classified as continuous or pulsed ASL (CASL or PASL, respectively). In CASL, continuous adiabatic inversion of spins is applied. In PASL, labeling is performed at once over a wide spatial range. Pseudocontinuous ASL (pCASL) has been introduced recently and uses a series of discrete labeling pulses. 1-8Despite its advantages, scanning time and reproducibility remain important issues in the clinical applicability of ASL. The use of higher-field strengths could overcome these issues because of increased signal-to-noise ratio, prolonged T1-weighted relaxation time of labeled blood, and better spatial and temporal resolution. 7,9 We hypothesized that scanning time of the CASL sequence can be reduced at 3T because fewer averages might suffice to obtain steady and reproducible CBF values. Our primary objective was to assess the number of averages needed to obtain steady and reproducible CBF values with use of 3T CASL.Also, we hypothesized that acquisition-related reproducibility of ASL will improve at higher-field strengths, whereas physiology-related reproducibility will not change. Previous CASL reproducibility studies were performed at 1.5T with test-retest timeframes of at least 1 hour. 4,6,10 Measurement of reproducibility within scans would be more valuable to assess physiologic variations. Our secondary objective was to assess 3T CASL reproducibility within scans (intrascan Ϸ 4 minutes), within sessions (intrasession Ϸ 10 minutes), and between sessions (intersession Ϸ 1 to 3 weeks). TechniqueAfter approval of the local ethics committee and written informed consent from all volunteers, we scanned 10 volunteers (5 men; age range, 25-33 years) without known cerebrovascular disease during 3...

Summary This study analyzed the mortality and causes of death in sickle cell disease patients in the Netherlands, to provide a baseline for monitoring the effect of the recently introduced neonatal screening programme and to indicate areas of improvement in the care for these patients. All children (<18 years) diagnosed with sickle cell disease in a tertiary hospital from 1985 to 2007 were included. Vital status was determined up to March 2008. A total of 298 children were included: 189 (63%) patients had HbSS, 17 (6%) HbSβ0 thalassaemia, 72 (24%) HbSC and 20 (7%) HbSβ+ thalassaemia. Twelve patients (4%) died during a total follow‐up of 3896 patient years. All known deaths were sickle cell disease‐related. Meningitis/sepsis (n = 4; 33%), stroke (n = 3; 25%) and death during a visit to the country of origin (n = 3; 25%) were the most common causes of death. The overall mortality rate was 0·27 deaths/100 patient years [95% confidence interval (CI): 0·15–0·43]. The estimated survival at the age of 18 years was 97·3% (95% CI: 95–99%). This report confirms that the burden of mortality in sickle cell disease is increasingly shifting to adults. It is recommended that compliance to antibiotic prophylaxis, thorough counselling and support for patients travelling abroad and specialized peri‐operative care should receive continuous attention.