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Resume But de l’etude Analyse de differents aspects psychologiques de la drepanocytose chez les sujets atteints. Plus specifiquement, nous nous interessons a l’impact de leur maladie, a l’adherence au traitement, aux emotions liees a leur maladie et a leur souhait d’education therapeutique. Patients et methode Nous soumettons l’adaptation du questionnaire « Brief Illness Perception Questionnaire » a des sujets atteints de 7 a 19 ans. Resultats L’echantillon compte 45 sujets, la majorite est issue du Congo. La moitie de nos sujets est issue de la premiere generation de migration. La majorite des sujets estime avoir une bonne sante. L’impact de la maladie sur leur quotidien a domicile est faible. Ils ont le sentiment de bien comprendre leur maladie. Globalement, ils ressentent des emotions variables en pensant a la drepanocytose. Ces emotions sont correlees a la migration. Ils associent difficilement au sujet de leur maladie. Conclusions La drepanocytose est vecue de maniere plutot positive. Les sujets severement atteints vont psychologiquement moins bien. De plus, la tristesse, la colere et la peur ressenties par rapport a la maladie sont correlees. La migration a un impact pejoratif important sur l’impact de la maladie chez nos sujets. La drepanocytose reste difficile a penser pour les sujets atteints. Il est capital de poursuivre le travail d’information societale, en Belgique et en Afrique.

Objective Red cell exchange transfusion is frequently used in the management of patients with sickle cell disease (SCD) either electively or chronically to maintain hemoglobin S (HbS)

Despite improvements in medical management, sickle cell disease (SCD) remains associated with severe morbidity and decreased survival. Allogeneic haematopoietic stem cell transplantation (HSCT) remains the only curative approach. We report the outcome of 50 consecutive children with severe SCD that received HSCT in our unit between November 1988 and April 2013. The stem cell source was bone marrow (n = 39), cord blood (n = 3), bone marrow and cord blood (n = 7) and peripheral blood stem cells (n = 1). All patients had ≥1 severe manifestation: 37 presented with recurrent vaso-occlusive crises/acute chest syndrome, 27 cerebral vasculopathy and 1 nephropathy. The conditioning regimen consisted of busulfan + cyclophosphamide (BuCy) before November 1991 and BuCy + rabbit antithymocyte globulin after that date. Since 1995, all patients have been treated with hydroxycarbamide (HC) prior to transplantation for a median duration of 2·7 years. Median age at transplantation and median follow-up was 8·3 and 7·7 years, respectively. Acute graft-versus-host disease (GVHD) and chronic GVHD were observed in 11 and 10 patients, respectively. An excellent outcome was achieved, with 8-year overall survival and event-free survival (EFS) rates of 94·1% and 85·6%, respectively. Since HC introduction, no graft failure occurred and EFS reached 97·4%. Prior treatment with HC may have contributed to successful engraftment.

The six-minute walk test (6MWT) was introduced in adults and children suffering from pulmonary or cardio-vascular conditions to assess their sub-maximal functional exercise capacity. In sickle cell disease (SCD), a reduced 6-minute walk distance was observed in adults with chronic pain, hip avascular necrosis and osteopenia ; and in children with low hemoglobin level, low fetal hemoglobin, a baseline elevated TRV. In a previous study (Dedeken et al., PLoS One 2014), we also showed that abnormal 6MWT was significantly associated with the presence of silent infarct. The aim of our study is to explore the evaluation of the 6MWT over time and to confirm the correlation with the cerebral vasculopathy in a larger cohort. This study was conducted at Hôpital Universitaire des Enfants Reine Fabiola (Brussels, Belgium) and included SCD children older than 6 years, regularly followed between 2011 and 2017 and who had at least two 6MWT. The age-standardized predicted value of the 6-minute walk distance (6MWD) was established as reported by Geiger. The 6MWT was considered as normal if the 6MWD was more than 80% of the age-standardized predicted value. Baseline hematological values, clinical events, cerebro-vascular disease, cardio-pulmonary parameters and disease-modifying treatment (DMT) were compared between those with normal and abnormal 6MWT and according to the 6MWD and between the 1st and the 2nd 6MWT overtime. 118 patients have been assessed twice and had at first evaluation a 6MWD of 90.6% (Range 49-119%), with an abnormal test found in 5.1%. The characteristics of the patients are detailed in the Table 1. The changes of the 6MWD and the biological data over time are detailed in Table 2. After 4 years of follow-up, 77.1% of patients were treated with Hydroxyurea (HU) and 16.6% patients were chronically transfused. In parallel with the increased HU prescribing rate, we have observed a significant increase of the Hb and the MCV and a decrease of reticulocytes and hemolysis parameters. The first 6WMT was performed at the median age of 10.3 years and the last one at the median age of 14.1 years. The median 6MWD increased over time including for non-chronically transfused patients. Girls performed less well in the 6MWT (93% for girls vs. 95.7% for boys; P = 0.03). Acute chest syndrome was significantly more frequent in boys (62%) compare to girls (38.7%). Nevertheless, no other differences were founded between boys and girls regarding biological values, clinical events or DMT. 26.5% of our patients have silent infarcts at a median age of 14.6 years. The 6MWD was the same in patients with and without silent infarcts (92.5% vs. 95% ; P=0.17) even when chronically transfused patients were excluded (94% vs. 95% ; P= 0.20). Patients with silent infarcts have a significant lower hemoglobin level and higher reticulocytes count, neutrophils count, LDH and MCV. In conclusion, the 6MWD observed in our cohort characterized by a very high rate of HU treatment is much higher than published in others series and improved over time. With only 5% of SCD patients having a 6MWD < 80% of the normal predicted value at last evaluation, we were not able anymore to confirm a correlation between the presence of silent infracts and abnormal 6MWT. Disclosures No relevant conflicts of interest to declare.

For centuries, writers have recorded their observations on pica. Nevertheless the association of pica with sickle cell disease (SCD) was poorly documented.Cross-sectional evaluation performed on SCD children and caregivers attending the outpatient clinic who were invited to complete questionnaires assessing behavior of pica.Out of 55 sickle cell children, 31(56.4%) reported practicing pica regularly. Substances ingested by patients covered a broad spectrum. Compared with the non-pica group, subjects who reported pica were younger and had lower hemoglobin (8.3 g/dl (7.6-9.7) vs. 9.1 g/dl (7.9-10.5): P0.01). The level of ferritin, zinc, copper, and lead was similar between the pica and non-pica groups (P0.05). Discussion In this series, there are many substances consumed by SCD children and adolescents, and we did not find an occurrence of similar substances among this select group. Pica children were younger and more anemic than non-pica patients.This study suggests that pica remains an unknown and under-reported clinical problem in children with SCD and seems to be related to the severity of anemia. The next step of this project aims to clarify causal mechanisms for pica and its association with SCD in a larger population.

Pain resulting from sickle-cell vaso-occlusive crisis (VOC) is often severe, prolonged and difficult to alleviate. Guidelines based on scientific evidence are lacking. In order to evaluate the effectiveness of our treatment protocol, we performed a population-based retrospective observational study on HbSS sickle-cell patients (n=22) admitted for severe VOC (n=48) during a 30-months period and managed with patient controlled analgesia (PCA). Median (10 th -90 th percentiles) visual analogical pain scale (VAS) at admission was 9.5(7–10). Patient received 0.3mg/kg (0.1–0.4) intravenous morphine at admission, then PCA was started with the following settings: continuous rate: 20 µg/kg/h (10–25), bolus: 25µg/kg (21–32), and 1.8 bolus allowed/hour (1.7–2.8). Six hours after admission, VAS was less than 7 in only 41% of cases. The median VAS declined steadily during hospitalization. Pain intensity was not correlated with morphine dosage. Success 6 hours after admission (VAS 2 CVO/year have the following characteristics: higher VAS at admission, higher morphine dosages, lower success rate and lower CRP, bilirubin, LDH and reticulocyte count. The difficulties encountered in the management of patients who experienced >2 VOC/year may be related to their genotypic particularities. For such patients, an increase of morphine dosage is required. We have developed a computer routine in order to reduce time and increase accuracy of PCA prescription, and to build a prospective database that enables continuous assessment of our treatment protocol.

4757 Early identification of sickle cell disease (SCD) by newborn screening (NS) is well established to be an efficient and practical tool in enhancing the health care of affected patients with SCD. The aim of our study, conducted in Brussels Region, was to assess whether there is an ongoing improvement of clinical outcome of children with SCD detected by the NS program. Universal NS was progressively implemented in Brussels starting within a few maternity wards in 1994 and extending to all maternity wards in 2000. Children identified with SCD progressively benefited from comprehensive expert medical care in three dedicated reference centers. Care included education, prevention, emergency and specific out-patient and in-patient treatments. To evaluate the improvement in comprehensive care, we reviewed data of children born from January 1st 2000 to December 31st 2003 (group A) and from January 1st 2005 to December 31st 2008 (group B). All data were recorded from January 1st 2000 to December 31st 2005 for group A and from January 1st 2005 to December 31st2010 for group B. Both groups had the same follow-up period accounting for 118 patient-years in group A and 259 patient-years in group B. Median follow-up was 3.5 yrs (range 2.06–5.83 yrs) and 4.1 yrs (range 2.08–5.96 yrs) in group A and B respectively. The major events such as septicemia, anemia, dactylitis, vaso-occlusive event (VOC), acute chest syndrome (ACS), symptomatic neurological events and hospital days were reviewed and compared during the study follow-up between the two groups. The reasons for hospitalization that were selected were: septicemia, pneumonia, urinary tract infection, osteomyelitis, gastroenteritis, VOC crisis, dactylitis, ACS, acute splenic sequestration, aplastic episodes and neurologic events. Several biological parameters were also reviewed. Among the 98 patients identified with SCD at birth, 33 (16 girls and 17 boys) and 65 (37 girls and 28 boys) belonged to group A and B, respectively. In group A, 25 children were HbSS, 2 HbSβ+ and 6 HbSC. In group B, 53 were HbSS, 5 HbSβ°, 5 HbSβ+ and 2 had an other genotype. Most of the patients developed at least one major adverse event during the study period. The proportion of patients having presented severe anemia and acute chest syndrome was significantly lower in group B than in group A ([table 1][1]). The higher rate of septicemia in group A could be due to the delayed implementation of national vaccination for Streptococcus pneumonia or to the poor prophylactic penicillin compliance. No difference was observed between both groups for dactylitis, VOC and clinical neurological event. No patient died during the study period. Hematological parameters at one year of age were not different between both groups. In conclusion, newborn screening is obviously recognized as a precious tool to identify patients with SCD. However, it must be part of a comprehensive care program. Our results demonstrated that its sustained effectiveness is really and clearly proven when it is coupled with a comprehensive and dedicated treatment program including close and regular parent education. This ongoing assessment should be performed to monitor and improve the screening program. Thereby the progressively implementation of comprehensive care has improved over time the quality of SCD management and then the outcome of patients in Brussels Region. | SCD related events | Group A (N=33) Follow-up from 2000–2005 Number of patients (%) | Group B (N=65) Follow up from 2005–2010 Number of patients (%) | P value | |:---------------------:| -------------------------------------------------------------- | -------------------------------------------------------------- | ------- | | Dactylitis | 9 (27.3) | 19 (29.2) | 1.0 | | Acute Chest Syndrome | 14 (42.4) | 10 (15.4) | 0.005 | | Vaso-Occlusive Crisis | 21 (63.6) | 29 (44.6) | 0.09 | | Anemia ≤ 6 g/dl | 22 (66.7) | 26 (40.00) | 0.02 | | Septicemia | 3 (9.1) | 1 (1.5) | 0.10 | | Neurological Event | 0 (0.0) | 2 (3.1) | 0.55 | Table 1. Patients identified by neonatal screening in Brussels with SCD related events Disclosures: No relevant conflicts of interest to declare. [1]: #T1

2107 [][1] The 6-minute walk test (6MWT) evaluates the sub-maximal functional exercise capacity and can be used together with the tricuspid regurgitant jet velocity (TRV) and pro-BNP to screen pulmonary hypertension in adults with sickle cell disease (SCD). A reduced 6-minute walk distance (6MWD) is observed in adults with SCD with chronic pain, hip avascular necrosis and osteopenia. In children with SCD, baseline elevated TRV is associated with a decline in age-standardized 6MWD. The aim of our study is to explore the submaximal exercise capacity of children with SCD followed at the Hopital Universitaire des Enfants Reine Fabiola, Brussels, Belgium and to analyze the factors affecting the 6MWT and the 6MWD. Since September 2011, all patients with SCD above 6 years of age were screened with the 6MWT as part of their follow-up in order to test if their functional capacity was altered. The age-standardized predicted value of the 6MWD was established as reported by Geiger. The 6MWT was considered as normal if the 6MWD was more than 80% of the age-standardized predicted value, moderately decreased between 60–80%, and severely altered less than 60%. Baseline hematological values, clinical events, cerebro-vascular disease, cardio-pulmonary parameters and disease-modifying treatment (DMT) were compared between those with normal and abnormal 6MWT and according to the 6MWD. Forty-six patients (20 boys and 26 girls) with a median age of 12 yrs were investigated. Forty-three were HbSS or HbSβ°, 2 HbSC and 1 HbSβ+. Thirty-two patients had a normal 6MWT and 14 an abnormal 6MWT. Only one patient had a severely altered test. These 2 groups were similar for age, sex, genotype and history of vaso-occlusive crisis or acute chest syndrome (ACS) as well as for the number of patients receiving DMT (either hydroxyurea (HU) or chronic transfusion). The proportion of patients with normal, conditional or abnormal transcranial doppler was also similar in both groups. Silent infarct (SI) on routine cerebral magnetic resonance imaging was found in 42.9% in the group with abnormal 6MWT versus only 19.4% in the group with normal 6MWT (p= 0.087). Pulmonary functional test, blood pressure, heart rate, systolic function and TRV were identical in both groups and only one patient had TRV >2.5m/sec. Baseline pulse oxymetry was slightly but significantly decreased in patients with abnormal 6MWT (98 versus 100%; p=0.022). Biological parameters were not statistically different between both groups. The 6MWD was not modified according to Hb, MCV, HbF, LDH and reticulocytes count or previous history of clinical event, except for the presence of SI ([Table 1][2]). Patients with or without SI were similar for age, sex, previous ACS or painful crisis as well as for hemolytic parameters (LDH: 945 versus 825 UI/l, p=0.832; reticulocytes: 273 versus 329 × 103/μl, p=0.548) and basal Hb (9.7 versus 8.8 g/dl, p=0.06). However patients without SI had significantly higher HbF and MCV values, and lower PMN count reflecting that most of them were treated with HU. In this cross-sectional study, the majority of children with SCD have a normal 6MWT. Abnormal 6MWT was not predicted by any clinical or biological features despite a trend to more SI in the group of children with abnormal test. In this series with only one high TRV patient, the sole factor which influences the 6MWD is the presence of SI. The lower exercise capacity of children with SCD with silent stroke may reflect some subclinical motor or sensitive impairment. Our data suggest also that HU might prevent SI which needs to be confirmed by larger prospective studies. | | Mean 6MWD in meters (SD) | p value | Mean Age in years (SD) | p value | |:------------------ | ------------------------ | ---------- | ---------------------- | ------- | | Hemoglobin (g/dl) | | · ≥ 9 (N = 24) | 531.5 (95.4) | 0.173 | 11.2 (2.8)

Abstract 4762 Introduction: Abnormalities of the clotting system are often observed in sickle cell disease (SCD). In addition to activation of coagulation, increased thrombin generation (TG) has recently been demonstrated in SCD children Aim of the study: To characterize the group of SCD children with increased TG in terms of clinical and biological parameters. Material and methods: TG was performed in the platelet-poor plasma of 97 SCD children at steady-state and 80 controls aged between 2 and 20 years. TG was triggered using 1 pM tissue factor and 4μM synthetic phospholipids with thrombomodulin to activate the protein C/protein S anticoagulant pathway. The Endogenous Thrombin Potential (ETP) and the peak height were analyzed. As these parameters increase with age in normal children, controls were distributed in 4 categories of age: [2–5], [6–10], [11–15] and [16–20] years. The mean ETP and the mean peak were calculated for each age category. A ratio for ETP (r ETP) and a ratio for the peak (r Peak) were defined for each control and patient as follows: individual ETP/ mean ETP and individual peak/ mean peak according to age category. Overall mean of r ETP and r Peak was then calculated such as to eliminate bias due to age. This calculated mean was 1.00 (0.39 – 1.61) for r ETP and 0.99 (0.28 – 1.81) for r Peak in controls. TG was considered abnormal if r ETP or r Peak value was above the mean + 2SD of controls (r ETP≥ 1.62 and r Peak≥ 1.82). Clinical and laboratory parameters were compared between SCD children having normal or increased ratios using the Mann Whitney test for numbers or the Fisher's exact test for proportions. P < 0.05 was considered significant. Results and Discussion: Overall 48 (49.4 %) patients showed either high r ETP or high r Peak whereas both parameters were increased in 31 (31.9 %) patients. As shown in Tables I and II, SCD children with elevated ratios were characterized by a younger age, shorter duration of hydroxyurea (HU) treatment, lower total hemoglobin level, higher reticulocyte and monocyte counts, higher LDH and D-dimer levels and a trend to increased procoagulant microparticle level (PMP) as compared with those having normal ratios. The higher D-dimer level in the group with abnormal ratios indicates that these children also manifest a higher degree of coagulation activation than those with normal ratios. Differences observed with markers of hemolysis are consistent with other reports suggesting a link between hypercoagulability and high hemolytic rate in SCD. The borderline significance of PMP is probably due to the use of exogenous phospholipids in TG which reduces the sensitivity of the test to endogenous phospholipids at the surface of PMPs. Conclusion: According to our study, Elevated Thrombin Potential is more frequently encountered in SCD children of younger age, with a shorter duration of HU treatment and with increased rate of hemolysis. These observations together with elevated D-dimer level seem to characterize SCD children with a hypercoagulable state. Disclosures: No relevant conflicts of interest to declare.

Abstract 2659 Sickle cell disease (SCD) has polymorphic manifestations, it is not well known by many physicians, and patients have often a precarious status. So despite enormous improvements in the understanding of the pathogenesis of SCD, patient care remains difficult.The objectives of this work were to create a clinical data base in order to learn the characteristics of this population, to create a network between practitioners (general, emergency and specialist practitioners), to provide state-of-the art and guidelines, it could be a tool to disseminate information, for education projects and for research; it could also represent a pilot project for other chronic diseases. A practical aspect of this data base is to improve follow-up and treatment of SCD patients by “online access from everywhere”. The SCD clinical data base was a national project and was approved by each local ethic committee; informed consent of each patient was obtained. The first step was to create the electronic database with several security measures (i.e. login, password, and separate administrators). The second step was to introduce patient's data. Patients were followed in different Academic and Secondary Care Centers. Data were collected from the initial contact until December 31, 2007. The data collection included parents' origin, hemoglobin phenotype, origin of diagnosis, clinical events, biological and radiological data, hospitalizations, and types of treatment. Up to date, we introduced 280 medical records (146 diagnosed by neonatal screening). The median age and follow-up of the cohort was 9.3 year (range, 0–44) and 6.5 year (range, 0–32), respectively. The first information provided was the predominance of patients from DR Congo (67.5 %), the occurence of severe events in 84 % of patients (Table 1), the predominance of Hb SS phenotype (90 %) and its severity, the report of septicemia which remain still very worrying (8.2 %), but also that clinical and radiological neurological events are sizeable, and that there is a good response to treatment intensification, particularly to bone marrow transplantation (BMT) and hydroxyurea. Table 1: SCD related events reported in the Belgian data base SCD related events Patients,%(n) Dactilytis 24.3 (68/280) Acute Chest Syndrome 18.9 (53/280) Recurrent Vaso-occlusive Crisis 61.8 (173/280) Anemia ≤ 6 g/dl 51.8 (145/280) Septicemia 8.2 (23/280) Splenic sequestration 7.9 (22/280) Stroke/TIA 3.9 (11/280) Osteonecrosis 5.0 (14/280) Osteomyelitis 2.5 (7/280) The main pathogen remained Streptococcus pneumoniae with no resistant strain despite regular prophylaxis. The second most common germ was Salmonella. Haemophilus influenza concerned older patients and disappeared since the introduction of the vaccination. The incidence of death was 2.86% (8/280). All of them were homozygous for Hb S. Two deaths occurred in the very early childhood due to the no compliance to antibio-prophylaxis in the first patient and poor follow-up in the second one. One death was very sudden after meningeal hemorrhage. Two other deaths happened in the adulthood, one after cerebral hemorrhage and the other one of unknown cause after going back to native country. The last three deaths were due to BMT complications (Table 2). Table 2: Causes of death reported in the national data base Patient Sex Phenotype Age at event Origin 1 F SS 18 m Cardiopulmonary arrest - Severe anemia 2 M SS 26 m Septic shock on St pmeumococcus septicaemia 3 M SS 7 y Secondary Leukemia after BMT 4 F SS 11 y 9 m Obliterans bronchiolitis/MOF* post BMT 5 M SS 14 y 3 m Meningeal hemorrhage 6 F SS 14 y 9 m Hemorrhage diathesis/MOF post BMT 7 F SS 18 y 9 m Cerebral aneurysm rupture 8 M SS 24 y 3 m Return to native country * Multiple Organ Failure In conclusion, the preliminary results confirmed the still high morbidity and mortality of SCD. It is not only a precious practical tool, but it also helps to improve clinical management of patients with SCD, it is a tool to identify risk factors and to tailor treatments. In this perspective, it constitutes a basis for prospective studies in view to validate criteria of disease severity and to adopt guidelines for adequate treatment. Disclosures: No relevant conflicts of interest to declare.

Abstract 4816 The objective of this study was to determine the efficiency of the neonatal screening program for SCD which was implemented in 1994 in few maternities and extended in 2003 to all maternities in Brussels. It is a systematic screening performed on liquid cord blood. Affected children are referred to a specialized center. We reviewed 146 medical records of patients with SCD born in Belgium and prospectively followed from the time of their diagnosis in three Brussels' Academic Centers. The study was approved by each local ethical committee and informed consent of each patient was received. Data were collected from the time of diagnosis of SCD (either done by neonatal screening or when a clinical event led to the diagnosis) until December 31, 2007. We focused on the subgroup of patients older than 3 years of age at December 31, 2007 and those were divided into two groups: those diagnosed by the neonatal screening (NS) and those diagnosed later (no NS). The incidence of major events (first septicemia, first stroke, first episode of severe anemia, first hospitalization and its duration, and death) was compared. Among the total population studied, 89 patients were diagnosed through the NS and 57 were not (no NS). While among those older than 3 years of age at the time of evaluation, 55 (median age 6.7 year, range: 3–16) and 49 (median age 11.2 year, range: 4–27) patients were in the NS or no NS group, respectively. The median age at diagnosis for the no NS cohort is 1 year (range: 1–6). The follow-up of the NS and no NS cohort account for 301.5 and 473.8 patient-years, respectively. Most of the patients were homozygous for Hb S (Hb SS) (82% in NS group and 94% in no NS group). Incidence of a first episode of septicemia was similar in both groups (10.9% in NS group versus 12.3% in no NS group). The median age at the time of sepsis was 27.6 months and 10 months in the NS and no NS group, respectively (Table 1). All the patients from the NS group were on penicillin prophylaxis versus 40 % in the no NS group. The main pathogen remained Streptococcus pneumoniae and there were no resistant strain despite regular prophylaxis.Table 1:Data on first septicemia in the non neonatal screening groupPatientAge at diagnosisAge at septicemiaProphylaxisPathogen13 m12 mYesSt. pneumoniae226 m3 mNoSt. pneumoniae310 m16 mYesSalmonella42 m14 mNoSt. pneumoniae511 m1 dNoSt.β hemol gr A61 m26 mYesSt. pneumoniae Incidence of stroke was 1.8% (1/55; 3.2 y.o.) in the NS cohort compared to 8.2% (4/49; 2.8, 5.3, 8.0 and 18.8 y.o.) in the no NS cohort. All the patients were Hb SS. The unique patient from the NS group was previously treated with Hydroxyurea (HU) for repeated vaso-occlusive crisis. 2 were also under HU prior to stroke for the same reason. Ischemic cerebral lesions were observed on MRI for all, except for the NS group patient. There was no significant difference either in the incidence of the first episode of severe anemia or the first hospitalization defined in term of number of days of hospitalization for both groups. Two deaths occurred in the NS cohort in the very early childhood (septicemia in one and acute severe anemia for the other). These deaths are attributable to no compliance to antibio-prophylaxis in the first patient and poor follow-up in the second one. Furthermore these deaths happen in the very early period after neonatal screening has been initiated. No death occurred in the NS group since 12 years probably due to better parents' education and comprehensive care. One death was observed in the no NS group (sudden rupture of cerebral aneurysm). In conclusion, neonatal screening program is feasible, safe and appropriate to detect SCD. It enables early diagnosis and therefore early treatment, and could improve both morbidity and eventually mortality of SCD. Although the relative small size of our study and the bias due to unreported early deaths by infection or severe anemia in the no NS group before the diagnosis of SCD has been done, our results are very encouraging: neonatal screening delays the age of the first severe infection and might reduce the incidence of early neurological complications. It also underlines the better outcome with improvement of parental education and comprehensive care. These data emphasize the need to continue neonatal screening for SCD in Brussels and to extend it to all Belgian maternities. Disclosures: No relevant conflicts of interest to declare.

Abstract 4821 Background: Red cell exchange transfusion is frequently used in the management of patients with sickle cell disease either electively or therapeutically to maintain an hemoglobin S (Hb S) level < 30–50%. This target is often difficult to maintain. In order to assess the effects of chronic partial exchange transfusion (CPET) a) on level of Hb and Hb S, b) on iron overload c) the need for chelation, d) on risk of long term adverse events and e) clinical outcome, we analyzed the data of sickle cell disease patients treated by long term CPET in our center. Methods/subjects: In the cohort of 163 SCD patients followed at University Children's Hospital at Brussels (Belgium), 10 benefit from CPET. Main reasons for CPET were neurologic disease (4), frequent ACS (3), previous severe hepatic cholestasis (2), leg ulcer (1) and pulmonary hypertension (1). The median age at start of treatment was 13 years (range 4 –19). These patients (6 males and 4 females) account for 248 exchanges during a median follow-up of 20 months (range 6– 36). These exchanges are until now performed manually and the volume exchanged is calculated taking into account the Hb level and the last HbS percentage. It is usually between 30 and 40 ml/kg BW. Except if severe anemia occurs, the goal of these exchanges is to keep a constant hematocrit level. All patients had a full red cell phenotype performed and received blood matched for ABO, Rhesus, Kell and Duffy antigens systems. The estimation of iron balance (iron intake- iron removed) was calculated yearly. Results: The pre-exchange Hb value was 9.5 g/dl (median; range: 7.7–10.9 g/dl) and the mean post value was 9.4/dl (range: 8.4– 11.1 g/dl). These values are not statistically different (p> 0.05). The majority of patients (9/10) are reached an HbS < 50% when measured 3–5 weeks after PET (just before the next procedure) with a median HbS value of 40% (range: 30–54). At start of CPET program, the median ferritin level was 439 ng/ml (range: 80 – 1704). Five patients had already a ferritin > 500 ng/mL due to numerous previous transfusions. At last evaluation, the median ferritin did not change significantly and was 531 ng/ml (range 84– 3840). The two patients with ferritin higher than 1000 ng/ml start chelation with good result for one. One The mean annual net RBC load were 1.72 ml RBC/kg/yr provided approximately 1.85 mg of iron/kg/yr. Individual data are given in table 1. CPET-treated patients exchanged showed clinical improvement with disappearance of SCD crisis and related complications. The procedure was well tolerated by most patients, and adverse effects were limited to mild hypotension (3/10). No autoimmune hemolysis or allo-immunisation was documented in this cohort. All children remained negative for HIV and hepatitis C virus infections. Conclusion: Manual CPET seems to be safe to prevent middle-term iron overload and the need of elation therapy in most of patients. CPET can therefore be recommended for SCD patients who required decreased in Hb S levels either prophylactically or therapeutically. Manual are safe, effective and easy to use when mechanized exchanges are not possible for technical reasons. Disclosures: No relevant conflicts of interest to declare.

Abstract 4807 Background: Pica defined as a compulsive and persistent intake of inedible substances or atypical food combinations at least one month. Association of pica and sickle cell disease (SCD) are poorly documented. Alerted by parents, we decided to verify systematically a) the occurrence of pica in SCD children b) to determine its duration, c) to identify the substances commonly ingested and d) to determine the characteristics among children and adolescents with SCD who reported practicing pica. Methods: SCD patients were seen in the outpatient clinic at the University Children's Hospital Queen Fabiola at Brussels between May 1, 2010 and July 30, 2010 as part of their regular follow-up. Data on sex, age, weight, height, body mass index, sickle cell phenotype, G6PD deficiency as well as biological data such as hematocrit mean corpuscular volume, fetal hemoglobin, plasma iron, zinc, copper, lead levels were recorded from their medical chart. Parents and patients were interviewed for the presence of eating disorders. Children and care givers completed questionnaires assessing symptoms of pica. Patients with acute illness, pregnancy, developmental delay, cognitive impairment or age younger than 3 years were excluded. Student t-test was used for difference in mean values groups by pica presence or absence. Fisher exact test was used to compare categorical variable. Difference in means values were considered statistically significant at p < 0.05. Results: Fifty-Five patients (24 males and 31 females) with a median age of 8. 9 years (6. 7– 11. 3) were evaluated during the study period. Forty-five patients (81.8%) originate from Central Africa, 10.9% (6/55) from West Africa, 1.8 %(1/55) from respectively East Africa, Italy and 3.6% (2/55) have mixed origin. Fifty –two (94.5%) patients are homozygous for Hb S (Hb SS), 3 are SC and 1 has Sβ° thalassemia. Thirty-one patients (56.4%) reported an history of pica and during the study period 29.1% (16/55) reported practicing pica regularly. The mean age of patient with pica was 7. 4 years significantly lower than non-pica patients (p 0.05) (Table 1). Iron status was identical. Lead overload was absent in the entire cohort. Hemolytic phenotype assessed by LDH was identical in both group and mild zinc deficiency was present in both groups. We did not observe difference due to hydroxyurée intake (p>0.05) Conclusion: In our study, pica appeared to have a very high prevalence in SCD children and adolescents. However, its etiology is still unknown. Except lower age and significant lower hematocrit value in pica patients when compared to non-pica ones, no differences in iron status, zinc deficiency or hematological parameters was found. The general health status evaluated by BMI was identical in both groups and the trend to more boys in the pica group is not significant. Further studies are necessary to establish the etiology of pica and its possible consequences on SCD. Disclosures: No relevant conflicts of interest to declare.