scholarly journals Successful Transition from Paediatric to Adult Care in Sickle Cell Disease: An Experience in a Belgian Center

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2495-2495
Author(s):  
Tracy Vandergraesen ◽  
Laurence Dedeken ◽  
Andre Efira ◽  
Phu Quoc Lê ◽  
Sophie Huybrechts ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Organ Damage ◽  
Transition System ◽  
Cell Disease ◽  
Adult Care ◽  
Walk Distance ◽  
The Us ◽  
Cardio Vascular

Abstract Introduction Despite reduction of mortality during childhood, young adults with SCD are at higher risk of morbidity and mortality after transition from paediatric to adult care, mainly in the US. To evaluate our transition system, we have studied the outcome of patients beyond the transition to adult care and looked if it was associated with an increase in vaso-occlusive crisis (VOC) and acute chest syndromes (ACS), the occurrence of progressive organ damage and changes in hydroxyurea prescription. Patients and Methods This study focus on sickle cell disease patients, born after January 1, 1980 and who have been transitioned from pediatrics (Hôpital Universitaire des Enfants Reine Fabiola, HUDERF) to adult care (CHU Brugmann), followed for minimum 2 years at HUDERF and having transitioned at least 2 years before December 2015. Demographic data, genotype, disease modify therapies, biological data, clinical data, acute clinical event, 6 minutes-walk test, cardiac echography) were collected retrospectively. Patients who underwent a hematopoietic stem cell transplantation were excluded from the analysis. Student test was used to analyse continuous variable and Wilcoxon regression for paired sample. Mc Nemar's test was used for comparison of paired nominal data and Chi-square test when data missed. Results The comparison of the data before and after transition is detailed in Table 1. Thirty eight patients (11 males) were included and made the transition at a median age of 20.1 years (range: 18.0-26.8). All patients were HbSS. The median age at diagnosis was 0.8 year (range: 0-7.95). The median duration of the follow-up before the transition was 15.95 years (range: 3.3 - 25.2) and after transition was 4.7 years (range: 2.2-5.8 years), accounting for 552.15 and 159.61 patient-years (PY) of follow-up respectively. Two patients died, 5.2 years and 0.6 year after the transition respectively. The death rate after transition was of 1.25/100 PY. Four women have delivered after a median gestation of 37.1 weeks (range: 35.9-38.7 weeks). The median new-born weight was 2.9 kg (range: 2.4-3.4 kg). The rate of disease modifying therapies remains stable after the transition. 33 patients were treated with hydroxyurea before transition compared to 35 after transition. Four patients were on chronic transfusion program before and 6 after transition. Hematological data were not statistically different at time of transition when compared to last follow-up, suggesting stable compliance to therapy but creatinine level increased significantly. VOC and ACS rate did not increased after transition as well as hospitalisation rate and days of hospitalisation. Tricuspid regurgitation velocity (TRV) increased significantly over the time but no patient developed pulmonary hypertension. The 6-minutes walk distance decreased significantly. Systolic and diastolic blood pressure increased significantly between the time of transition and the last follow-up. Regarding the educational status (8 data missing), 21 (70%) patients were able to reach graduate school and only 4 were not able to finish high school. Conclusion Our data provide useful information on the outcome during the years after transition. No increase in hospitalizations, VOC or ACS was observed. This is very different from the data provided by Blinder et al. One of the reason could be that comparing to the US situation, the Belgian Health Care System provide access and quality of healthcare even in underprivileged population. The prescription rate of HU remained unchanged and the compliance to treatment evaluated by hematological parameters remained stable. Nevertheless, changes in cardio-vascular parameters and creatinine levels suggest that despite the well-standardized follow-up for young adult patient with sickle cell disease, progressive organ damage leading to renal and cardio-vascular disease might not be prevented. However, compared to the literature, we have found less high level of TRV (9.7% vs. 24-45%), no patient with pulmonary hypertension (compared to 6-10%). In addition, our patients seems to have a better 6-minutes walk distance even in the childhood cohort (paediatrics data : 562m vs. 491m in Waltz study; adults data : 490m vs. 398m). Additional data from a larger number of patients as well as a longer period of follow-up are required to confirm the efficacy of our transition system. Disclosures No relevant conflicts of interest to declare.

scholarly journals Implementation of an Educational Intervention to Optimize Self-Management and Transition Readiness in Adolescents with Sickle Cell Disease

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3536-3536
Author(s):  
Cecelia Calhoun ◽  
Regina Abel ◽  
Hai Anh Pham ◽  
Shomari Thompson ◽  
Allison A King
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Psychosocial Development ◽  
Sexual Development ◽  
Independent Living ◽  
Self Management ◽  
Cell Disease ◽  
Adult Care ◽  
Transition Readiness

Abstract Background: The transition from the pediatric setting to adult care is a challenge for many adolescents with chronic disease. Patients with sickle cell disease (SCD) represent a unique cohort as the timing of psychosocial development of adolescence often coincides with worsening end organ damage. Previously, we used the Adolescent Autonomy Checklist (AAC) modified to include SCD specific tasks that patients with SCD need to practice in order to transition to adult healthcare and independent living. This study sought to use the AAC to measure the effects of skill based educational handouts on improving self-management and transition readiness in adolescents with SCD. Methods: This was a single center, retrospective study approved by the Washington University Institutional Review Board. Inclusion criteria were patients with SCD, age 13-21 years, and completion of pre and post assessments. As standard care, patients from a pediatric hematology clinic completed the AAC-SCD. The AAC-SCD assesses skill level in twelve domains (Table). The tool includes 100 items, and users check "can do already" or "needs practice" for each item. After review with the coordinator, participants were given skill-based handouts based on up to five noted deficits. Patients completed the AAC-SCD at the subsequent clinic visit. In addition to baseline and follow up AAC-SCD data, medical and demographic data were collected via chart abstraction. All data were entered into SPSS for statistical analysis, including descriptives, paired sample T-tests, and bivariate Pearson's correlations. Results: A total of 61 patients completed baseline and follow up. Of those participants, 49.2% were female. The mean age was 15.4 (+ 2.2) years. The genotypic distribution was as follows: 67.2% HbSS, 19.7% HbSC, 3.3% HbS-beta-thal+ and 9.8% HbS-beta-thal0. The majority of patients received healthcare coverage via Medicaid (52.5%), private insurance (45.6%) and 1.6% had no insurance coverage. Twenty-five patients (42.0 %) had a history of stroke or silent cerebral infarct and 34 (55.7%) were currently taking or were previously prescribed hydroxyurea. Formal academic support (IEP or 504 Plan) was reported for 20 (32.8%) of patients. At baseline, patients needed the most help with skills in the kitchen, housekeeping, personal care and leisure. Statistically significant improvements (p< 0.05) occurred in skills related to laundry, housekeeping, healthcare, sexual development and living arrangements. Modest sized and statistically significant correlation between the receipt of the educational handouts and decreased number of items marked "needs help" occurred in the areas of money management (r=-0.27, p=0.044), vocational skills (r=-0.27, p=0.046;) and laundry (r=0.32, p=0.015). A post hoc analysis by age groups 13-15 (n= 34),16-18 (n=24) and 19-21 (n=3) showed a decreased amount of items marked "needs help" in the areas of sexual development for both 13-15 year olds (r=0.42, p=0.024) and 16-18 year olds (r=0.93, p=0.001) as well. Conclusion: Transition skills improved over time among adolescents with SCD. While we cannot say for certain if gains in knowledge occur with age as development progresses or if a formal transition program can be credited, providing educational materials on transition related skills within a clinic setting was associated with significant improvements in three of the domains. Our preliminary data offers insight into what skill deficits may be most amenable to educational interventions based on age group. As is the case with medical management, the development of a multimodal intervention is needed to prepare adolescents with SCD to transition to adult care and independent living. Clinic based education is a simple intervention that could be one component of future approaches to transition. Disclosures No relevant conflicts of interest to declare.

Utilization and Costs of Deferoxamine in Patients with Thalassemia, Sickle-Cell Disease, or Myelodysplastic Syndrome Receiving Frequent Transfusions.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 5583-5583
Author(s):  
Thomas E. Delea ◽  
May Hagiwara ◽  
Simu K. Thomas ◽  
Jean-Francois Baladi ◽  
Pradyumna D. Phatak ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Drug Acquisition ◽  
Iron Chelator ◽  
Cell Disease ◽  
Total Cost ◽  
The Us ◽  
Health Insurance Claims ◽  
Using Data

Abstract Background. Patients with thalassemia, sickle-cell disease (SCD), and myelodysplastic syndromes (MDS) receiving frequent transfusions require chelation therapy to prevent complications of iron overload. Deferoxamine (DFO) is an effective iron chelator that has been shown to reduce the morbidity and mortality associated with transfusional hemosiderosis. Data on the utilization and costs of DFO treatment are limited however. The objective of this study was to document the utilization and costs of DFO therapy in patients with transfusion-dependent anemias seen in typical clinical practice. Methods. Retrospective, observational study using data from large health insurance claims database spanning 1/97–12/04 (“study period”) and representing approximately 40 million members enrolled in &gt;70 health plans across the US. Study subjects included members meeting the following criteria: ≥1 claims with diagnosis of thalassemia (282.4x), SCD (282.6x ), or MDS (ICD-9-CM 238.7x); ≥8 claims (on different days) for a transfusion of whole blood or red cells; ≥2 claims (on different days) for DFO. Follow-up was defined as the period from the date of first DFO claim (“index date”) to end of study period, disenrollment, or 15 days after last claim for DFO, whichever occurred first. Outcomes included the number of claims for DFO and grams of DFO dispensed and the costs of DFO therapy, including costs of drug acquisition and administration. Outcomes were analyzed by qualifying diagnosis, numbers of transfusions received, and grams of DFO dispensed. Results. We identified 155 subjects who met all inclusion criteria, including 35 with thalassemia, 68 with SCD, and 52 with MDS. On average, patients received one transfusion every 3.4 weeks of follow-up. Mean DFO grams dispensed were 306 per year. MDS patients received the most transfusions but the least DFO. Only 38% of MDS patients received ≥3 g of DFO per week (≥156 g per year). Mean total DFO costs were $18,025 annually ($10,217 for drug and $7,808 for administration). Controlling for other factors, utilization of DFO was not associated with number of transfusions received; administration costs were only weakly associated with amount of DFO received. Thalassemia SCD MDS All Values are Mean±SD N 35 68 52 155 Follow-up, days 612 ± 481 420 ± 403 274 ± 336 414 ± 418 Age, years 19 ± 12 17 ± 11 63 ± 11 33 ± 24 Transfusions per year 15 ± 7 12 ± 4 24 ± 13 16 ± 10 DFO claims per year 29 ± 34 41 ± 46 30 ± 20 34 ± 37 DFO grams per year 311 ± 233 343 ± 243 223 ± 234 306 ± 241 Cost DFO acquisition, $ per year 10,287 ± 8,264 11,625 ± 8,339 7,293 ± 7,543 10,217 ± 8,207 Cost DFO administration, $ per year 7,674 ± 11,503 9,109 ± 8,177 5,403 ± 5,649 7808 ± 8,438 Total cost of DFO, $ per year 17,961 ± 17,047 20,734 ± 12,114 12,696 ± 10,886 18,025 ± 13,348 Conclusion: In this population of frequently transfused patients (mean 16 transfusions per year), utilization of DFO was low (mean &lt;1 gram per day) suggesting inadequate chelation. Costs of DFO administration were high, representing approximately 43% of the total cost of chelation.

Comparing Patterns for Transitioning the Care of Young Adults with Sickle Cell Disease Versus Hemophilia: The Toronto Experience

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 2072-2072 ◽  
Author(s):  
Ewurabena Simpson ◽  
Richard Ward ◽  
Melanie Kirby ◽  
Isaac Odame
Keyword(s):  
Health Care ◽  
Young Adults ◽  
Sickle Cell Disease ◽  
Medical Care ◽  
Disease Severity ◽  
Sickle Cell ◽  
Cell Disease ◽  
Adult Care ◽  
Effective Transfer

Abstract Abstract 2072 Background: The Hospital for Sick Children (HSC) in Toronto, Canada cares for more than 700 children with sickle cell disease (SCD), which is the largest Canadian population of children with SCD. Since 2009, the SCD Program at HSC has partnered with adult hematologists within the Red Blood Cell Disorders program at Toronto General Hospital (TGH) to develop a coordinated strategy for transitioning the care of young adults with SCD. Hemophilia is a chronic hematological condition which, like SCD, has a spectrum of disease severity that requires multidisciplinary follow up. At HSC, we care for nearly 200 patients with hemophilia A and B and have a long-established partnership with adult hematologists, which has led to an effective transfer of patients with hemophilia into adult care. In Ontario, adult health providers are remunerated according to a fee-for-service billing schedule. In contrast, pediatric subspecialists are mainly salaried under an alternate funding plan. Until 2010, adult hematologists who provided medical care for individuals with hemophilia received a significantly higher pay scale than those who cared for individuals with SCD. This was changed in July 2010 so that adult hematologists receive commensurate remuneration for services rendered for both hemophilia- and SCD-related medical care. Objectives: 1. To compare the patterns for transitioning patients of varying disease severity within the pediatric and adult SCD and hemophilia programs in Toronto, Ontario. 2. To identify barriers and enablers that have influenced the transition of young adults with SCD within a universal health care system. Methods: Data for active, transitioned and inactive patients in the HSC and TGH clinical programs are maintained in a database at HSC. These patient numbers were gathered according to sickle cell genotype. Similar data were available for hemophilia patients who were transitioned from HSC to adult care. Chi-square analyses were used to compare the proportions of patients in the sickle cell and hemophilia programs that were transitioned between 2009 and 2011. Results: Conclusion: The HSC-TGH- partnership has significantly reduced the number of youth with SCD who continue to be followed at HSC or are lost to follow up. However, a significant number of young adults within the HSC SCD program still need to be transitioned to adult care. For the sustainable expansion of this transitional care strategy, health policymakers must collaborate with tertiary and community level health care providers to develop a coordinated and distributed strategy for the effective delivery of comprehensive health care services for young adults with SCD. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Optimal disease management and health monitoring in adults with sickle cell disease

Hematology ◽  
2019 ◽  
Vol 2019 (1) ◽  
pp. 505-512 ◽  
Author(s):  
Jo Howard ◽  
Swee Lay Thein
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Multidisciplinary Care ◽  
Organ Damage ◽  
Cell Transplant ◽  
Cell Disease ◽  
Adult Care ◽  
Pharmacological Agents ◽  
Hematopoietic Stem ◽  
Age Related

Abstract In countries with access to organized health care, survival of children with sickle cell disease (SCD) has greatly improved, resulting in a growing population of adults with SCD. Transition from pediatric to adult care presents many challenges for the patient, who now faces the reality of emerging complications in many organs that are cumulative, adding to other age-related nonsickle conditions that interact and add to the disease morbidity. We recommend regular comprehensive annual assessments, monitoring for early signs of organ damage and joint clinics with relevant specialists, if applicable. While maintaining a low threshold for intervention with disease-modifying therapies, we should always keep in mind that there is no single complication that is pathognomonic of SCD, and nonsickle comorbidities should always be excluded and treated if present. We need to reevaluate our approach to managing adults with SCD by putting a greater emphasis on multidisciplinary care while proactively considering curative options (hematopoietic stem cell transplant and gene therapy) and experimental pharmacological agents for adults with SCD of all ages before complications render the patients ineligible for these treatments.

Hemolysis-Associated Elevation in Tricuspid Regurgitation Velocity Predicts Reduction in Six-Minute Walk Distance After Two Years of Follow up in Children and Adolescents with Sickle Cell Disease.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 574-574
Author(s):  
Victor R. Gordeuk ◽  
Lori Luchtman-Jones ◽  
Andrew D. Campbell ◽  
Sohail R Rana ◽  
Mehdi Nouraie ◽  
...  
Keyword(s):  
Steady State ◽  
Sickle Cell Disease ◽  
Children And Adolescents ◽  
Sickle Cell ◽  
Cell Disease ◽  
Walk Distance ◽  
Six Minute Walk Test ◽  
Minute Walk Distance ◽  
Minute Walk

Abstract Abstract 574 Background. Elevated tricuspid regurgitation velocity (TRV) as determined by echocardiography correlates with elevated systolic pulmonary artery pressure and is associated with increased morbidity and mortality in adults with sickle cell disease. The importance of elevated TRV in children and adolescents with sickle cell disease is not known. The Pulmonary Hypertension and the Hypoxic Response in SCD (PUSH) study is an ongoing, longitudinal and observational multicenter study of children with sickle cell disease. Methods. Baseline echocardiography and six-minute walk test were performed prospectively in 361 children and adolescents with sickle cell disease at steady state and then repeat studies were performed in 209 after a median of 22 months of follow up (range 10 months to 36 months), also at steady state. A hemolytic component was derived by principal component analysis of baseline values for reticulocyte count, lactate dehydrogenase, aspartate aminotransferase and total bilirubin. Results. TRV or six-minute walk test were measured at both baseline and follow-up in 193 patients. Twenty-one of these 193 patients had elevated TRV of 2.60 m/sec or higher at baseline. Elevated baseline TRV was associated with high hemolytic rate in 15 patients, defined as hemolytic component above the median for the population studied, and with lower hemolytic rate in six patients. Elevated baseline TRV with high hemolytic rate predicted elevated TRV at follow up (odds ratio 7.7; 95% confidence interval [CI] 2.5 to 24.2; P <0.001) but elevated baseline TRV with lower hemolytic rate did not (odds ratio 1.6; 95% CI 0.2 to 14.1; P = 0.7). Elevated baseline TRV with high hemolytic rate also predicted a decline in the six-minute walk distance by 10% or more at follow-up (hazard ratio 3.9; 95% CI 1.4 to 10.7; P = 0.009). In contrast, higher cardiac output as measured by the left ventricular end diastolic dimension z-score was associated with reduced risk for a decline in the walk distance (hazard ratio 0.7; 95%CI 0.6 to 0.9; P = 0.006). Conclusion. Steady-state TRV elevation in association with a high hemolytic rate occurs on screening in about 8% of children and adolescents with sickle cell disease and is predictive of elevated TRV and reduced six-minute walk distance after approximately two years of follow. Such children may be at risk for adverse clinical consequences of pulmonary hypertension as young adults. Further studies are indicated to identify the molecular mechanisms and to develop appropriate medical management for children and adolescents with this complication. Disclosures: No relevant conflicts of interest to declare.

Systematic Evaluation Of Chronic Organ Damage In Adult Sickle Cell Patients. A Seven-Year Follow-Up Study

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 4683-4683
Author(s):  
Charlotte F.J. van Tuijn ◽  
Marein Schimmel ◽  
Eduard J. van Beers ◽  
Bart J. Biemond
Keyword(s):  
Pulmonary Hypertension ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Outpatient Clinic ◽  
Organ Damage ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
Avascular Osteonecrosis ◽  
Tertiary Teaching

Introduction The occurrence of organ damage in sickle cell disease (SCD) is a crucial determinant for both medical treatment and prognosis. In a previous study, we systematically analyzed the prevalence of SCD related organ damage and complications in adult sickle cell patients in a tertiary teaching hospital in the Netherlands. We now describe a seven-year follow-up of this patient cohort, to provide an insight into the course of the various forms of organ damage and SCD related complications. Methods At baseline in 2006, 110 adult patients visiting the outpatient clinic of our hospital were enrolled. All enrolled patients from the primary analysis were included for follow-up. Patients were screened for sickle cell related manifestations during visits to the outpatient clinic biannually. Various forms of sickle cell related organ damage and complications (presence of microalbuminuria, renal failure, pulmonary hypertension retinopathy, iron overload, cholelithiasis, avascular osteonecrosis, leg ulcers, acute chest syndrome, stroke, priapism and admissions for painful crises) were routinely screened according to international guidelines. Patients with genotype HbSS/HbSβ0 and HbSC/HbSβ+were grouped for further analysis. Results Of all originally included patients (N=110), nine were lost to follow-up (N=9). The mean age of the current study cohort is 37 years (IQR 27-46). Overall, 59 patients (58%) developed a new form of organ damage or new complication since baseline analysis, including eight patients who deceased (7 due to a sickle cell disease related death). In the HbSS/HbSβ0 genotype group (N=60) we found an increase in the percentage of patients who have had an Acute Chest Syndrome (29% to 47%) or have been diagnosed with avascular osteonecrosis (15% to 24%), retinopathy (23% to 34%) or pulmonary hypertension (31% to 48%). In the HbSC/HbSβ+ (N=35) group we found an increase in the occurrence of avascular osteonecrosis (9% to 14%), retinopathy (59 % to 70%) and pulmonary hypertension (9% to 19%). Furthermore, the use of hydroxycarbamide increased in both genotype groups and the frequency of admissions for painful crises remained stable for both genotype groups. Conclusion In the past period of seven years 58% of the patients in a previously well descript cohort of adult SCD patients developed a new sickle cell related complication. For some forms of organ damage or complications a substantial increase occurred dependent of a patient’s genotype. Disclosures: No relevant conflicts of interest to declare.

Survival into Adulthood in Sickle Cell Disease from the Dallas Newborn Cohort

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 559-559 ◽  
Author(s):  
Melissa D. Mathias ◽  
Song Zhang ◽  
Zora R. Rogers ◽  
George R. Buchanan ◽  
Alecia C. Nero ◽  
...  
Keyword(s):  
Overall Survival ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Early Mortality ◽  
Cell Disease ◽  
Adult Care ◽  
Clinical Encounters ◽  
Lower Baseline ◽  
Baseline Hemoglobin

Abstract Background: The natural history of sickle cell disease (SCD) includes premature death. Advancements in care, such as penicillin prophylaxis, have made death during childhood uncommon. Prior survival estimates for adults with SCD have been limited by survival bias (subjects enrolled as adults) or the limitations of administrative data. No prior study has estimated survival for adults with SCD from a newborn cohort. Herein, we report an continued observation of subjects in the Dallas Newborn Cohort (DNC)1. Methods: The DNC is an inception cohort of patients with SCD defined by: (1) diagnosis by Texas newborn screening after October 31, 1983; (2) evaluation in the pediatric sickle cell program at UT Southwestern (UTSW) / Children’s Medical Center Dallas (CMCD); and (3) confirmation of SS, SC, Sβ0 or Sβ+ genotype. Patients followed at CMCD are transitioned to adult care before the 19th birthday. To identify transitioned patients or those lost to follow-up (LTFU), we reviewed the medical records at Parkland Memorial Hospital (PMH) and UTSW University Hospital (UH), the public and private hospitals affiliated with CMCD and the most common care-sites for transitioned patients. In addition, for patients no longer actively followed at CMCD, we queried the National Death Index (NDI). The NDI is a centralized database of death records obtained from all state vital statistic bureaus in the US and is maintained by the National Center for Health Statistics. As of our query in April 2014, deaths occurring from 1/1/79 - 12/31/11 were available in the NDI. Probabilistic matching is performed by the NDI to identify subjects using identifiers such as name, social security number, birthdate, gender, state of birth, and race. Patients who were LTFU or transitioned to adult care were censored at 12/31/11 or the date of their last clinical encounter, whichever occurred latest. Patients actively followed at CMCD, PMH, or UH were censored at 12/31/13. In addition to descriptive statistics, we constructed survival curves using the Kaplan-Meier method to evaluate overall survival and survival by genotype. Also, Cox Proportional Hazard modeling was used to assess genotype, baseline hemoglobin, baseline reticulocyte count (%), and baseline oxygen saturation as potential predictors of early mortality. To test the sensitivity of the NDI to identify deceased individuals, we submitted 36 known deaths from the cohort. Results: The DNC is now comprised of 1214 patients with 16,636 years of follow-up. The cohort is 49.2% female with a mean age, and duration of follow-up, of 13.2 years (Range 0.1 – 30.0 years). SCD genotypes included 60.5% HbSS, 30.6% HbSC, 6.8% HbSβ+, and 2.1% HbSβ0. Of the 1214 patients in the cohort, 511 were active in the pediatric program, 195 were in the pediatric age range but had moved or were LTFU, 238 were adults with 1 or more visits at PMH or UH, 234 were adults with no subsequent identifiable clinical encounters, and 36 were deceased. Of 36 known deaths submitted to the NDI, 34 were identified by their databases (94.4% sensitivity). Of the 606 patients who were LTFU or aged out of the pediatric program, only 10 were reported to have died by the NDI. One of them was confirmed to be erroneous because the patient had clinical encounters after the NDI-reported date of death. In addition to these 9 deaths in the NDI, 8 additional patients were known to have died as of 12/31/13, bringing the total number of deaths to 53 (4.3%). Of these, 21 (39.6%) have been identified since the last cohort update in 2007. Overall survival in the DNC to age 25 years was 91.4% (95% CI: 88.4%, 93.7%) for all members (Figure), 87.7% (95% CI: 83.3%, 91%) for those with HbSS or HbSβ0, and 98.6% (95% CI: 96.7%, 99.4%) for those with HbSC or HbSβ+. In univariate analysis, early mortality was associated with SS or Sβ0 genotypes (compared to HbSC or HbSβ+), lower baseline hemoglobin, and low baseline SpO2 but not gender or reticulocytes. In multivariate analysis, early mortality was associated with only lower baseline hemoglobin (Hazard Ratio 0.76 [95% CI 0.59, 0.98], P=0.037). Conclusions: These are the first modern estimates of survival into adulthood with an unbiased newborn cohort of SCD patients. Early mortality is associated with low baseline hemoglobin. Survival to age 20 remains excellent at 95% but the mortality rate appears to accelerate in early adulthood. Reference: 1. Quinn et al. Blood. 115(17):3447-52, 2010. Figure 1 Figure 1. Disclosures No relevant conflicts of interest to declare.

scholarly journals High Incidence and Recurrence Rate of Venous Thromboembolic Events in Patients with Sickle Cell Disease

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 2381-2381
Author(s):  
Bart J. Biemond ◽  
Ewout R. Egner ◽  
Erfan Nur ◽  
Charlotte F.J. Van Tuijn
Keyword(s):  
Risk Factors ◽  
Sickle Cell Disease ◽  
Recurrence Rate ◽  
Sickle Cell ◽  
Organ Damage ◽  
Clinical Risk Factors ◽  
Cell Disease ◽  
Clinical Risk ◽  
High Incidence

Abstract Introduction: Sickle cell disease (SCD) is characterized by chronic hemolytic anemia and chronic inflammation resulting in endothelial damage, neutrophil and platelet activation, enhanced cell adhesion and coagulation activation. Due to these conditions, sickle cell disease is considered to be a procoagulant condition. However, limited studies have evaluated the incidence of thrombosis in sickle cell disease and clinical risk factors for thrombosis are not clear. Here we present a retrospective cohort study to determine the incidence of VTE in a well characterized cohort of adult sickle cell patients. The aim of the study was to assess the cumulative incidence of venous thromboembolic events in patients with SCD and to relate this complication with clinical risk factors, genotype, organ damage and laboratory parameters. Methods: All patients with SCD (HbSS, HbSC, HbSβ0 thalassemia, HbSβ+ thalassemia) of ≥ 18 years in a tertiary clinic for SCD at the Amsterdam University Medical Center in the Netherlands were eligible for the study. Patients with a history of prior VTE or VTE or VTE at first presentation were excluded. Patients were divided into a subgroups with a severe genotype (HbSS/HbSβ0 thalassemia) and a mild genotype (HbSC/HbSβ+ thalassemia). Hospital electronic records were analyzed for occurrences of deep vein thrombosis (DVT) or pulmonary embolism (PE) objectified with diagnostic tests (ultrasound, high probability ventilation/perfusion scan or CT angiography). All sickle cell related complications, organ damage and clinical risk factors at the time of the VTE were scored. General laboratory parameters were gathered from patient records in steady state condition. Results: In total 228 patients were included in the study with a mean age of 24 ± 10 years at the start of follow up years and a total follow up of 1548 patient years. Median follow up was 5 years (IQR 2-10). Twenty-one patients suffered one or more VTE episodes (9.2%), resulting in an incidence rate in the entire cohort of 13.6 VTE events per 1000 patient years (95%Cl 9.7-22.0). We recorded 8 recurrences of VTE in 5 patients (1 patient suffered two VTE recurrences, and 1 patient had three VTE recurrences) with a median time to recurrence of 3 years (IQR 1-4). Mean age at the time of first VTE was 29 years. The first VTE episodes consisted of an isolated DVT of the leg/arm in 8/21 patients, an isolated PE in 10/21 patients, and a combined DVT and PE in 3/21 patients. Of these first VTE episodes 9/21 (43%) were found to be idiopathic, 5/21 (24%) occurred during oral contraceptive use and 7/21 (33%) were provoked by recent surgical procedure/hospital admission/central venous catheter. VTE was significantly associated with a previous history of acute chest syndrome (ACS) (OR 10.6 [3.3 - 46.6] P<.001), avascular necrosis (AVN) (OR 5.6 [2.1 - 15.0] P <.001) and a ferritin level >1000 μg/L (OR 3.8 [1.4 - 10.2] P=0.023). No significant association with other forms of organ damage was found. Patients with a severe genotype had a higher incidence of VTE than patients with a mild genotype (11.4% versus 5.6%). In the severe genotype subgroup, lower median HbF levels correlated with an increased risk of VTE (P <.005). Ten patients died during follow-up at a mean age of 40 ± 16 years. Conclusion: Sickle cell patients have a remarkably high incidence rate of VTE (13.6 events per 1000 patients years) with a high recurrence rate (23.8%). This confirms the hypercoagulable state of patients with SCD. Sickle cell related complications like ACS and AVN were associated with VTE. Given the high incidence and recurrence rate, awareness for VTE in SCD patients is warranted and long term anticoagulation may be indicated. Disclosures No relevant conflicts of interest to declare.

scholarly journals Real-World Clinical Burden of Sickle Cell Disease in the US Community-Practice Setting: A Single-Center Experience from the Foundation for Sickle Cell Disease Research

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 5856-5856
Author(s):  
Lanetta Bronté-Hall ◽  
Matthew Parkin ◽  
Courtney Green ◽  
Elsa Tchouambou ◽  
Lynn Huynh ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Real World ◽  
Research Funding ◽  
Organ Damage ◽  
Community Practice ◽  
Cell Disease ◽  
End Organ Damage ◽  
The Mean

Background Sickle cell disease (SCD) is a progressively debilitating monogenic disease characterized by unpredictable, acute, life-threatening episodes and chronic complications such as hemolytic anemia and end-organ damage. It presents with a range of severity resulting in significant morbidity, poor quality of life, and early mortality. Real-world data on treatments and clinical outcomes for patients (pts) with SCD are limited, particularly in the community clinical care setting. The objectives of this retrospective real-world study were to characterize clinical manifestations and management of pts with SCD treated at the Foundation for Sickle Cell Disease Research (FSCDR). Methods A retrospective longitudinal analysis of an electronic health records (EHR) database from the FSCDR, which captured laboratory testing, treatments, and records on outpatient, emergency, and inpatient visits, was conducted. All unique pts with SCD assigned a medical record number in the EHR (N=172) were considered. Data from external records were manually entered into the EHR to supplement the EHR database. To address limitations of a real-world database, medical records for all pts included were manually reviewed and validation was performed on 10% of the sample. Pt demographics, clinical characteristics, hydroxyurea (HU) treatment (as captured by prescriptions and usage notes), administration of red blood cell transfusions, vaso-occlusive crisis (VOC) events and acute chest syndrome (ACS) (based on physician assessment) were described. Annual VOC rates were summarized by dividing the number of events by the total follow-up duration in years. Results In total, 122 pts with SCD were included. Twenty-three pts were excluded as they were not actively seeking care and/or had no relevant clinical data from 01/01/15-07/19/19. Data for 27 pts <22 years of age were unavailable at time of analysis (results of this cohort will be included in the presentation). Among all pts, 76 (62.3%) were female and 118 (96.7%) were Black or African American. The mean age at time of analysis was 39.1 (standard deviation [SD] 12.3) years with 9 (7.4%) 22-24 year-olds, 67 (54.9%) 25-40 year-olds, 35 (28.7%) 41-55 year-olds, and 11 (9.0%) ≥ 56 year-olds. The most common genotypes were HbSS (77.0%) and HbSC (17.2%). Over a mean follow-up period of 2.9 (interquartile range: 2.0, 4.4) years, 27 (22.1%) pts were treated with HU and 66 (54.1%) pts received transfusions, of which 1 (1.5%) pt chronically received episodic transfusions (i.e., continuous monthly transfusions for ≥ 6 months). The mean total hemoglobin (Hb) was 8.3 (SD 1.7) for HbSS pts and 10.9 (SD 1.5) for HbSC pts. Among pts who never received HU or transfusions during follow-up, mean Hb was 9.4 (SD 1.7) for HbSS pts and 11.4 (SD 1.6) for HbSC pts. Among pts who ever received HU or transfusions, mean Hb was 7.7 (SD 1.3) for HbSS pts and 10.3 (SD 1.1) for HbSC pts. Eleven (9.0%) pts had ≥ 1 ACS whereas 97 (79.5%) pts had ≥ 1 VOC event. ACS and VOC events occurred mostly in pts 25-55 years of age (ACS: 9 [81.8%] pts aged 25-40 years and 2 [18.2%] pts aged 41-55 years; VOC: 54 [55.7%] pts aged 25-40 years and 29 [29.9%] pts aged 41-55 years). Annual rates of VOC are described in Figure 1. Conclusions This is one of the first studies to describe clinical characteristics and management of pts with SCD in a community practice setting. Higher Hb levels among pts who never vs ever received HU or transfusions during follow-up may be a reflection of who were selected for treatment and additional studies to take into account timing of treatment and Hb assessments and confounding factors need to be conducted. The study found high VOC rates particularly among pts aged ≤ 40 years. Lower VOC rates among older pts do not necessarily indicate less severe disease. One potential reason for lower VOC rates among older pts with SCD is that cumulative exposure to ischemia-related tissue injury and resulting end organ damage may decrease VOC-related pain over time. Further investigation to elucidate the rate of VOC decline observed in older pts is needed, including looking at underlying health and end organ damage. While limitations are inherent in real-world studies, these findings underlie the ability to and importance of studying SCD management and clinical outcomes in community care settings. This study highlights the clinical burden of SCD and possibly higher than expected unmet need in this community setting. Disclosures Bronté-Hall: bluebird bio: Research Funding. Huynh:bluebird bio: Research Funding. Puri-Sharma:bluebird bio: Employment. Chang:bluebird bio: Research Funding. Chawla:bluebird bio: Employment. Signorovitch:bluebird bio: Research Funding.

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