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This study&rsquo, s objective was to assess, on a national scale, residual risks of death, major disease-related events, and quality of care during the first five years in children diagnosed at birth with sickle cell disease (SCD). Data were retrospectively collected from medical files of all children with SCD born between 2006&ndash, 2010 in France. Out of 1792 eligible subjects, 1620 patients (71.8% SS or S/beta&deg, thalassemia -SB&deg, ) had available follow-up data, across 69 centers. Overall probability of survival by five years was 98.9%, with 12/18 deaths related to SCD. Probability of overt stroke by five years in SS/SB&deg, patients was 1.1%, while transcranial Doppler (TCD) was performed in 81% before three years of age. A total of 26 patients had meningitis/septicemia (pneumococcal in eight cases). Prophylactic penicillin was started at a median age of 2.2 months and 87% of children had received appropriate conjugate pneumococcal vaccination at one year. By five years, the probability of survival without SCD-related events was 10.7% for SS/SB&deg, patients. In contrast, hydroxyurea was prescribed in 13.7% and bone marrow transplant performed in nine patients only. In this study, residual risks of severe complications were low, probably resulting from a good national TCD, vaccination, and healthcare system coverage. Nonetheless, burden of disease remained high, stressing the need for disease-modifying or curative therapy.

Background: The goals of newborn screening programs (NBS) for sickle cell disease (SCD) are to reduce early mortality and morbidity, by introducing preventive measures like penicillin prophylaxis, pneumococcal vaccines and Transcranial Doppler (TCD) screening. The main objective of this study (NCT 03119922) was to assess on a national scale and during the first 5 years residual risks of death, overt stroke and bacterial meningitis/septicemia resulting from current TCD and pneumococcal prophylactic utilization in SCD children diagnosed at birth. An additional objective was to determine the frequency of other main SCD-related events and the use of disease-modifying or curative therapy. Methods: Using the national NBS database between 2006-2010, data was collected in 2014/2015 from the patients' medical files up to the age of 5 years, and included age at first prescription of penicillin, occurrence of death/overt stroke/bacterial meningitis and septicemia, age at first transfusion (TF), use of pneumococcal vaccines/chronic transfusion program/hydroxyurea (HU)/hematopoietic stem cell transplantation (HSCT) and TCD results. Results: Out of 1792 eligible subjects, 152 (8%) were lost to follow-up. A total of 1620 patients with available follow-up data across 69 centers constituted the EVADREP cohort. Of them, 71.8% had SS or S beta°-thalassemia (SB°) while 20.3% had SC /S Beta+-thalassemia (SB+) disease, and 59.6% resided in Paris area. Overall mortality rate for all patients was 0.23/100 person-years during the 5 first years of life and probability of survival at 5 years was 98.9% (95%CI: 98.2-99.3), with 18 deaths during the study period, 12 related to SCD (11 patients SS or SB°). The probability of overt stroke at 5 years was 1.1% all in SS/SB° patients. DTC was performed in 56% and 81% of patients before 2 and 3 years of age, respectively. The probability of abnormal TCD at 5 years was 10.4%. A total of 26 patients had a severe infection (meningitis or septicemia), lethal in 8 cases and caused by a pneumococcal strain in 8 cases. More than 99% of patients were prescribed prophylactic penicillin, started at a median age of 2.2 months of life. Full pneumococcal vaccination (at least 4 PCV and one P23) was performed in only 47.4% before 3 years but the probability of receiving P23 was 90% at 5 years. At 3 years, 42.9% of the EVADREP cohort had experienced a first VOE and the probability of survival without VOE was 62.4% [60.0-64.8] at 5 years. When pooling all SCD-related events (death, stroke, severe infection, VOE, acute anemia, as well as abnormal DTC, pneumonia and/or ACS and transfusion), 58.6% of children had experienced at least one event at 3 years. The probability of survival without SCD-related events at 5 years was 10.7% [0.9-12.6] for SS/SB° patients and 46.3% (41.5-51) for SC/SB+. In sharp contrast, HU was prescribed in 13.7% and HSCT performed in 9 patients only. Vaccination and DTC coverage, use of TF program and HU were significantly higher in larger centers. Summary/Conclusion: Current residual risks of severe complications and mortality rates on a national scale are similar to those observed in SCD expert center cohorts, presumably as a result of good TCD and vaccination coverage and an easy access to health care in France. Further improvement could be achieved with better referral to centers of expertise. The burden of disease is still high and increased use of disease modifying or curative therapy should benefit more children in the future. Disclosures Brousse: Add medica: Consultancy; bluebird bio: Consultancy. Bernaudin:BlueBirdBio: Consultancy; AddMedica: Honoraria, Other: Help for travel; GBT: Membership on an entity's Board of Directors or advisory committees. de Montalembert:AddMedica: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; bluebird bio, Inc: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees.

Background Pediatricians taking care of sickle cell children in France are concerned about giving travel advice. Very few articles are published and no study has been done about it. A lot of pediatricians are using their own experience to decide if sickle cell children can travel abroad. Studying the consequences of such travel for sickle cell children is important to discuss common recommendations. Methods We conducted a prospective study from June 2006 to December 2007 on desires to travel expressed during our consultations with sickle cell children. We studied notable events that occurred during travel and at least 2 months after return. Results Of 52 desires to travel, 10 were cancelled. All of the 42 trips were to Africa. Median duration of travel was 1.29 months (0.5–3). Median age at travel was 7.6 years (0.2–17.7). Events during travel were two hospitalizations (4.8%), a transfusion (2.4%), and four paramedical or medical examinations (9.6%). After return, four events occurred: two SS children had Plasmodium falciparum malaria (4.8%) and two had digestive bacteremia (4.8%) in SC and Sβ+ children. No event occurred during plane travel. None of our patients died. Conclusions The primary risk for sickle cell children traveling to Africa is infection: malaria first and digestive septicemia second. These risks are increased by long travel and poor sanitary conditions. Each travel should be prepared a long time before departure, and each pediatrician should insist on malaria prophylaxis and sanitary conditions, especially for young children. Trips should be shorter than 1 month when possible. A longer prospective study will be done to confirm these results.

Résumé Un groupe de médecins généralistes et multidisciplinaire de spécialistes contribue à définir les différentes formes de sinusites ou rhinosinusites aiguës de l'enfant à partir des principaux symptômes et signes cliniques. Le rôle des pathologies associées telles que l'allergie, les troubles immunitaires est envisagé. L'incidence, la présentation clinique et la prise en charge des complications sont présentées. Les indications des examens radiologiques et biologiques sont analysées. La prise en charge médicale en particulier, la place et le type des antibiotiques sont discutés. Des propositions de prise en charge selon des situations cliniques rencontrées en pratique sont présentées.

We report a case of congenital nephrotic syndrome with acute renal failure associated with multiple organ injury requiring peritoneal dialysis. Maternal serology, including hepatitis B, HIV, and Treponema pallidum hemagglutination (TPHA) and the Venereal Disease Research Laboratory (VDRL) tests were all negative at 10 weeks of gestational age (GA). At 34 weeks of gestation, the ultrasound examination showed hydrops fetalis. The umbilical cord puncture found anemia, with hemoglobin at 8.4 g/dl, thrombocytopenia at 43,000/mm, cytomegalovirus (CMV)-polymerase chain reaction (PCR) and parvovirus-PCR were negative. The child was born at 35 weeks of gestational age and immediately transferred to an intensive care unit because of septic shock. The clinical findings were sclerema neonatorum with blisters, hepatomegaly, splenomegaly, and macroscopic hematuria. The initial blood analysis showed hemoglobin 11 g/dl, thrombocytopenia 22,000/mm (despite intrauterine blood and platelets transfusion), reticulocytes >150,000/mm, C-reactive protein (CRP) 233 mg/L, urea 4 mmol/L, serum creatinine 72 μmol/L, proteins 56 g/L, serum glutamic oxaloacetic transaminase (SGOT) 482 UI/L, serum glutamic pyruvic transaminase (SGPT) 224 UI/L, haptoglobin 600 μg/dl. Symptomatic treatment was initiated by albumin, anticoagulation, and recombinant erythropoietin. At the 9th day of life, CRP was 51 ng/ml, and hemoculture showed Staphylococcus epidermidis, motivating vancomycin treatment. Serum creatinine increased concomitantly to 262 μmol/L.

Relieving pain effectively in children with painful sickle cell crisis (PSCC) is a challenging clinical problem. Notwithstanding analgesic treatment, some hospitalized children for a PSCC seem to continue in pain. Objectives: To describe the evolution of pain in hospitalized children receiving analgesics for a PSCC. Methods: Retrospective study of all one-year hospitalizations for PSCC in a pediatric university hospital. Pain assessments were retrieved from patients' records. The minimum, mean and maximum pain scores per day of hospitalization were determined for each patient. Results: From May 2008 to April 2009, 156 admissions for PSCC occurred. The median (interquartile; extremes) age of admitted patients was 9.9 (5.1-13.5; 0.3-18.3) years. The mean (SD; extremes) duration of hospitalization was 3 (2.8; 0-17) days. The mean (SD; extremes) pain score on arrival to the ward was 3.9 (3.3; 0-10). During the hospitalization period the intensity of pain decreased progressively from day 1 to day 7, thereafter there was an increase in the intensity of pain followed by a decrease on day 14. Figure shows the distribution of maximum pain scores for each day of hospitalization. Conclusion: Daily maximum pain scores are high (>5/10) for about a quarter of children during the first 6 days of hospitalization for PSCC notwithstanding analgesic treatment. Improvement of current analgesic protocols seems necessary.

1365 Does the Pain Scale Type Influence the Assessed Pain Intensity in Painful Sickle Cell Crisis in the Pediatric Emergency Department?

Objectives: To describe (1) the severity of painful sickle cell crisis (PSCC) in children presenting to the ED and (2) the ED analgesic management. Methods: Retrospective study of all one-year visits to an ED for a PSCC. Results: From May 2008 to April 2009, 189 visits concerning 87 children were registered. In 149 (79%) visits, an initial pain score was recorded. Assessments were carried out with four different scales. A 0-10 Visual Analog Scale was used in 97 (65.1%) of assessments; 3 other scales were used for the remaining 52 (34.9%) assessments. In order to uniform all the assessment data, the latter were transformed into a 0-10 scale. The median (interquartile) pain score of all assessments was 6 (4-8). The 149 pain assessments were divided into three groups: mild pain (≤ 4/10 score) including 40 (26.8%) assessments, moderate pain (5-7/10) including 61 (40.9%) assessments and severe pain (≥ 8/10) including 48 (32.2%) assessments. The frequency of use of step 1 WHO analgesics (acetaminophen, ibuprofen), step 2/a codeine, step 2/b nalbuphine, and step 3 morphine were, respectively, 60%, 7.5%, 75%, and 0% for mild pain, 60.7%, 8.2%, 73.8%, and 1.6% for moderate pain, and 52.1%, 2.1%, 60,4% and 8.3% for severe pain. Nitrous oxide/oxygen was used for all venipunctures and in 38.3% of visits to alleviate persistent PSCC. In 124 of 149 (83%) visits, children were admitted to the hospital. Conclusion: In 1/3rd of ED visits for PSCC, children were in severe pain. Morphine was rarely used even for severe pain.

Background: Acute splenic sequestration (ASS) is a life threatening event in SCD during early childhood. Subsequent related mortality has been greatly reduced by both parental education and appropriate management. Moreover SCD screening in neonatal period has allowed early detection contributing to the decrease in fatal attacks. Objectives: Our aim was to update splenic sequestration epidemiology in a French cohort followed after neonatal screening. Methods: we reviewed the medical files of SCD children born between 2000–2007 followed since birth in 5 pediatric centres. We included all SCD children diagnosed with acute splenic sequestration, defined as an acutely enlarging spleen with a fall of hemoglobin (Hb) concentration of at least 2g/dL. Results: A total of 266 episodes of ASS occurred in120 children. There were 69 boys and 51 girls. Genotypes were as follows: 9 S beta° thalassemia, 2 SD-Punjab, 4 SC, 105 SS. In the SS subgroup, median age at first episode was 16 months [1–83] and 34,8% of these first episodes occurred before 12 months of age. Associated symptoms were found in 37 cases (isolated fever n=20, vaso occlusive crisis n=5, identified viral or bacterial infection n= 12) and did not influence the rate of recurrence. Mean Hb level during first crisis was 5.2g/dL [3.3–7]. Mean age at first episode for those who experienced only one episode (n=43) versus more than one episode (n=62) was statistically different (18 months (+/−10) versus 28 (+/−22); p=0.002, Student test). Median interval between first and second episode was 3.5 months [1–31]. 31 patients had 3 or more episodes with a mean interval between second and third interval of 2 months [1–17]. There was no significant difference in the mean age at first episode in children who experienced 2, 3 or more episodes. There was only one fatal case due to acute anemia which occurred in a girl at first recurrence (Hb:2,2g/dL). After the second attack 14(22.5%) patients were splenectomized, 26 patients (41.9%) were started on a transfusion program (followed in 13 cases by splenectomy) and 21 were followed up. Splenectomy was performed at a median age of 4.5years. Conclusion: These results show that ASS remains a major concern in the management of SCD children. We show a high rate of recurrence and young age at first episode favours this recurrence threat. This study further confirms the effectiveness of early diagnosis and parental education in the decrease of mortality. Prospective studies on spleen dysfunction and additional research on predictive factors are warranted.