scholarly journals Vaso-Occlusive Events in Pediatric Sickle Cell Disease: Quantifying Social Disadvantage and Its Impact on Hospitalizations

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 1949-1949
Author(s):  
Marti Goldenberg ◽  
Meghan McCormick ◽  
Kristine Ruppert ◽  
Enrico M Novelli ◽  
Ram Kalpatthi
Keyword(s):  
Medical Home ◽  
Hospital Readmission ◽  
Pediatric Patients ◽  
Socioeconomic Disadvantage ◽  
Acute Chest Syndrome ◽  
Preventative Care ◽  
Cell Disease ◽  
Disadvantaged Group ◽  
Pain Scores

Abstract Background: Sickle Cell Disease (SCD) has established socioeconomic disadvantage resulting in increased healthcare utilization. Among the pediatric population, the implementation of preventative care measures within the medical home model has improved outcomes and lifespan. Area deprivation index (ADI) is an established method for quantifying socioeconomic disadvantage and has been shown to be associated with increased hospital readmission in adults and pediatric patients with chronic disease (Singh Am J Public Health. 2003, Kind et al. Ann Intern Med. 2014). ADI has been applied to adults with SCD and vaso-occlusive events (VOE) but has not been investigated in pediatric patients. We explored the role of ADI in pediatric patients with SCD admitted with VOE in order to characterize disadvantage and the hospitalization characteristics that impact hospital readmission. Methods: This retrospective review included 675 consecutive emergency department and hospital admissions for VOE among 101 pediatric (≤ 21 years old) patients with SCD from 2016-2019 at a single urban, US-based medical center. One hospital admission for each patient was selected at random. Information extracted included demographics, SCD characteristics, admission complications, entry and discharge pain scores, length of stay, and management characteristics. Variables were compiled in a descriptive table and compared in logistic regression models against a primary outcome of 7-day readmission. The 2018 ADI dataset was used to assign an ADI value based on the census block corresponding to the patient-reported zip-code for the specific hospital admission (https://www.neighborhoodatlas.medicine.wisc.edu). ADI values were grouped by most and least deprived with higher scores (6-10) indicating more deprivation. Summary statistics described patient and disease characteristics in these groups. Results: Within our cohort, the state and national ADI was calculated for 101 patients with a median age of 14 years (50.4% female; 98.0% Black) (Table 1). Most patients were publicly insured (80.2%). The Hb SS genotype was the most common genotype, followed by Hb SC (22.7%). Median ADI rank was 9 with 11 patients (10.9%) classified as less disadvantaged (ADI 1-5) and 90 patients (89.1%) as more disadvantaged (ADI 6-10). We found the less disadvantaged group had even numbers of public and private insurance use while 83.3% of the patients in the more disadvantaged group had public insurance. The Hb SS genotype was more common in the less disadvantaged (90.9%) than the more disadvantaged (74.4%), where Hb SC was overrepresented (24.4%). Greatest prevalence of mental health disease and hospital complications, including acute chest syndrome, avascular necrosis, and pneumonia, were observed in the more disadvantaged. Additionally, the more disadvantaged group included a greater number of patients with scheduled follow-up (61.1%) and shorter time to follow-up (median: 31 days). For treatment and management, both groups had high numbers of prescriptions for hydroxyurea (HU) and opiates, with slightly higher numbers in the less disadvantaged group (82% HU; 100% opioids) compared to the more disadvantaged (61% HU; 81% opioids). Length of hospital-stay and pain scores were similar across groups. The 7-day readmission rate was 9% for the less disadvantaged group and 14% for the more disadvantaged group. Conclusions: We applied a validated measure of socioeconomic disadvantage to a group of pediatric patients with SCD and VOE. Our cohort consists of more disadvantaged patients across ADI scales. Readmission rate was low for both groups and there was no relationship between greater ADI scores and hospital readmission. Within the more disadvantaged group, the higher prevalence of mental health illness as well as associated disease complications may have been countered by protective factors including established follow-up, shorter time to follow-up, and high number of prescriptions for hydroxyurea and opiates. Our results are consistent with the protective effect of ADI on recurrent acute chest syndrome in pediatric SCD patients (Alishlash et al. Pediatr. Blood Cancer. 2021) highlighting the importance of preventative care within the SCD medical home. Further research is warranted on identifying key protective factors that may reduce acute care utilization and social disparity in this population. Figure 1 Figure 1. Disclosures Novelli: Novartis Pharmaceuticals: Consultancy.

scholarly journals Quantifying Social Disadvantage for Patients with Sickle Cell Disease: Implications for Quality of Care

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 317-317
Author(s):  
Nicole Savidge ◽  
Susan K Parsons ◽  
Daqin Mao ◽  
Ruth Ann Weidner ◽  
Kimberly S Esham ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Healthcare Utilization ◽  
Research Funding ◽  
Readmission Rate ◽  
Socioeconomic Disadvantage ◽  
Census Block ◽  
Cell Disease ◽  
Disadvantaged Group ◽  
Genetics Research ◽  
Pain Scores

Abstract Background: Socioeconomic disadvantage negatively affects healthcare utilization and disease outcomes. The Area Deprivation Index (ADI) is a well-established method for quantifying socioeconomic disadvantage that combines 17 US Census block indicators of poverty, education, housing, and employment (Singh Am J Public Health. 2003). ADI scores have been shown to be associated with hospital readmission and mortality in the general population and in chronic diseases, such as diabetes (Kind et al. Ann Intern Med. 2014). However, the ADI has not been used in patients with sickle cell disease (SCD), a group that faces health disparities based on race and socioeconomic status and that has high healthcare utilization, including a 41% readmission rate among young adults (Brousseau et al. JAMA. 2010). We applied the ADI to patients with SCD, hospitalized for vaso-occlusive crisis (VOC). We described patient, disease, and treatment characteristics by high and low ADI scores. Methods: This retrospective cohort study includes 449 consecutive hospitalizations for VOC among 63 adult patients (≥18 years) with SCD from 2013-2016 at an urban, US-based academic medical center. For this analysis, one hospitalization was randomly selected for each patient. Demographics, including street address, SCD characteristics, complications, treatment, hospital entry and discharge pain scores, length of stay and 30-day readmission were abstracted from electronic medical records (EMR) by trained study staff. History of SCD complications (e.g., acute chest syndrome, avascular necrosis) were reviewed by two hematologists. The 2013 Massachusetts (MA) ADI dataset was used to assign patients an ADI decile value based on the census block corresponding to the patient-reported address in the EMR (https://www.neighborhoodatlas.medicine.wisc.edu/). ADI deciles were calculated using ADI scores from all census blocks in MA ranked from lowest to highest (1-10), where higher scores indicate more deprivation. ADIs were divided into a more disadvantaged group and a less disadvantaged group at the sample median similar to previous research (Hu et al. Am J Med Qual. 2018). Summary statistics described patient and disease characteristics separately by these groups. Results: Out of the 63 patients in our cohort, the ADI was calculated for the 57 patients who had a valid MA address (90.5%). The median age was 26 years with 56.1% female and 77.2% black (Table). The majority of patients were publicly insured (28.1% Medicare, 66.7% Medicaid), while only 5.3% were privately insured. The most common genotype was Hemoglobin (Hb) SS (61.4%), followed by Hb SC (22.8%). The median MA ADI rank was 6; 27 patients (47.4%) were classified as less disadvantaged and 30 (52.6%) as more disadvantaged. Among the less disadvantaged group, 88.9% were black and 11.1% were Hispanic, while the more disadvantaged group was 66.7% black and 30.0% Hispanic. The less disadvantaged group had fewer Medicaid patients (59.3%) than the more disadvantaged group (73.3%). Hb SS genotype was more common in the less disadvantaged group (70.4%) than the more disadvantaged group (53.3%), where there were more patients with Hb SC (30.0%). For treatment and management, the less disadvantaged group had more patients prescribed hydroxyurea (74.1%) and home opioids (92.6%), compared to the more disadvantaged group (56.7% hydroxyurea; 80.0% home opioids). Length of hospital stay and pain scores were similar across the two groups. The 30-day readmission rate was 14.8% for the less disadvantaged group and 23.3% for the more disadvantaged group. Conclusions: We successfully applied a validated measure of socioeconomic disadvantage to a cohort of hospitalized patients with SCD. The more disadvantaged group consisted of more Hispanic patients with less Hb SS genotype and more Medicaid insurance. Further, there appeared to be differences in SCD management and readmissions by group, indicating possible disparities and opportunities to improve care and outcomes. Based solely on a patient's address from the EMR, the ADI can be used at the point of care to identify and ensure that the most vulnerable patients are receiving appropriate levels of social support. Given that SCD is referred to as a disease of double disadvantage, it is crucial to understand the intersection of this debilitating chronic illness within the context of socioeconomic disadvantage. Disclosures Parsons: Seattle Genetics: Research Funding. Rodday:Seattle Genetics: Research Funding.

scholarly journals Evaluation of Vaso-occlusive Crisis Management With Patient-Controlled Analgesia in Children With Sickle Cell Disease Requiring Hospitalization

2021 ◽  
Vol 26 (6) ◽  
pp. 615-623
Author(s):  
Claire Arbitre ◽  
Yves Pastore ◽  
Benoit Bailey ◽  
Niina Kleiber ◽  
Nancy Robitaille ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
Patient Controlled Analgesia ◽  
Pain Scores ◽  
A Prospective Study ◽  
Reduced Time ◽  
Median Pain

OBJECTIVE The aim of this study was to review the use of patient-controlled analgesia (PCA) in sickle cell disease (SCD) for pediatric patients with vaso-occlusive crisis (VOC) in our institution and to compare the effect of early vs late PCA start on pain relief and LOS. METHODS This retrospective study included all pediatric patients treated with PCA for a severe VOC from 2010 to 2016. “Early-PCA” was defined as start of PCA within 48 hours of arrival. Time to reach adequate analgesia was defined as the time to reach 2 consecutive pain scores less than 5/10 at 4-hour interval. RESULTS During the study period, 46 patients presented 87 episodes of VOC treated with PCA. Sixty-three patients with VOC were treated with Early-PCA and 24 with Late-PCA. Both groups were comparable except for median pain score at admission; the Early-PCA group had higher scores: 9.0/10 vs 7.0/10. Time to reach adequate analgesia could be evaluated only in a subset of patients (n = 32) but was shorter in the Early-PCA group with a median difference of 41.0 hours (95% CI −82.0 to −6.0). Early-PCA was associated with a median reduction in LOS of 3.4 days (95% CI −4.9 to −1.9). There was no difference between the 2 groups in terms of side effects and occurrence of acute chest syndrome during hospitalization. CONCLUSIONS In this study, a reduced time to reach adequate analgesia and LOS was noted in the Early-PCA group for severe VOC. A prospective study is required to confirm these results.

Accuracy of Point-of-care Lung Ultrasonography for Diagnosis of Acute Chest Syndrome in Pediatric Patients with Sickle Cell Disease and Fever

2016 ◽  
Vol 23 (8) ◽  
pp. 932-940 ◽  
Author(s):  
Dina D. Daswani ◽  
Vaishali P. Shah ◽  
Jeffrey R. Avner ◽  
Deepa G. Manwani ◽  
Jessica Kurian ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Point Of Care ◽  
Acute Chest Syndrome ◽  
Lung Ultrasonography ◽  
Cell Disease

NT-Pro Brain Natriuretic Peptide Levels and the Risk of Stroke and Death in the Cooperative Study of Sickle Cell Disease.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 1541-1541 ◽  
Author(s):  
Roberto F Machado ◽  
Mariana Hildescheim ◽  
Laurel Mendelsohn ◽  
Gregory J Kato ◽  
Mark T Gladwin
Keyword(s):  
High Risk ◽  
Sickle Cell Disease ◽  
Incidence Rate ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Cooperative Study ◽  
Cell Disease ◽  
Risk Of Death ◽  
History Of

Abstract Abstract 1541 Poster Board I-564 Background Elevations in NT-proBNP levels are associated with hemolysis-associated pulmonary hypertension and mortality in adults with sickle cell disease. The association of this vasculopathy with the risk of stroke has not been explored. The Cooperative Study of Sickle Cell Disease (CSSCD) is the largest cohort of adult and pediatric patients with sickle cell disease with the longest median follow-up. Using stored plasma samples from patients enrolled in the CSSCD, we tested the hypothesis that adult patients with high levels of NT-proBNP would be at a high risk of death and stroke and that pediatric patients with a high BNP might be at a high risk of stroke. Methods A threshold NT-pro-BNP value previously identified to predict mortality in adults with sickle cell disease was used to determine the association between the risk of stroke and mortality in a cohort of 758 participants (pediatric cohort, n=428 and adult cohort, n=330) in the CSSCD. Results The prevalence of an abnormal NT-proBNP level ≥ 160 pg/ml was 27.6 % in patients in the adult CSSCD cohort. The prevalence of a NT-proBNP level ≥ 160 pg/ml was 27.4 % in the pediatric cohort, which is at least in part explained by known higher normal NT-proBNP levels in children, especially during the first year of life. The incidence of the development of a high NT-proBNP level in subjects with a normal baseline level was 6 % over a mean follow-up time of 5.9 years (incidence rate per 100-person years of 1.03) in the pediatric cohort and 16.5 % over a mean follow-up time of 1.9 years in the adult cohort (incidence rate per 100-person years of 8.59). In subjects with normal baseline levels, multivariate logistic regression analysis of clinical and laboratory factors associated with the development of a high NT-proBNP level included increasing age (OR 3.43, 95% CI 1.6-7.2, P = 0.001), low hemoglobin (OR 0.47, 95% CI 0.3-0.7, P < 0.001), high white blood cell count (OR 1.69, 95% CI 1.05-2.7, P = 0.03), high creatinine (OR 2.22, 95% CI 1.1-4.3, P = 0.02), blood urea nitrogen (OR 1.64, 95% CI 1.2-2.3, P = 0.004), alanine aminotransferase (OR 1.26, 95% CI 1.01-1.6, P = 0.04) and uric acid levels (OR 2.32, 95% CI 1.4-3.8, P = 0.001), and history of leg ulcers (OR 7.46, 95% CI 2.0-28.5, P = 0.003). Elevated NT pro-BNP levels were associated with indices of hemolytic anemia (hemoglobin: OR 0.33, 95% CI 0.2-0.4, P < 0.001) and a history of stroke (OR 1.86, 95% CI 0.9-3.7, P = 0.02). An NT-pro-BNP level ≥160 pg/ml was a predictor of mortality (RR 6.24, 95% CI 2.9-13.3, P < 0.001) and stroke (RR 3.84, 95 % CI 1.0-14.3, P = 0.05) in this cohort. Conclusions A high NT-proBNP level is a major risk factor for death in patients with sickle cell disease. These findings provide further support for a mechanistic link between hemolytic anemia and the development of cardiovascular complications in this patient population. Finally, we have identified a novel widely available biomarker of the risk of death and stroke in sickle cell disease. Disclosures No relevant conflicts of interest to declare.

A North American Experience Of Hemoglobin SC Disease, Its Complications, and Management

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 2221-2221 ◽  
Author(s):  
Veronique Naessens ◽  
Richard Ward ◽  
Kevin H.M. Kuo
Keyword(s):  
Sickle Cell Disease ◽  
Avascular Necrosis ◽  
Sickle Cell ◽  
Care Center ◽  
Comprehensive Care ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
Baseline Hemoglobin ◽  
Hemoglobin Sc Disease

Background The phenotype of hemoglobin SC (HbSC) disease is distinct from sickle cell anemia (SCA) (HbSS and S/b0) but management of adults is mostly derived from studies of the latter group. Longitudinal observational studies on the complications and outcomes of hemoglobin SC disease are largely confined to centers outside North America. The unique ethnic composition of our cohort, consisting of mostly Western Africans and West Indians, permits further characterization of the HbSC phenotype. Objective to describe the baseline characteristics and long-term complications of a cohort of adult HbSC patients followed in a Canadian sickle cell comprehensive care center. Methods A retrospective observational cohort study was conducted on all adult patients with HbSC disease attending a sickle cell comprehensive care center in Toronto, Canada from 1994 to 2013. Baseline demographics, acute and chronic complications attributable to sickle cell disease, and laboratory data were collected. Medians were used to describe continuous variables, while percentages or ratios for categorical variables. Logistic regression was used to examine factors influencing the main clinical complications. Results 104 patients were included in the analysis, comprising of 38.5% males and 61.5% females. Median length of follow-up was 3.5 years (1 - 19) and median age at last recorded visit was 35 years (18 - 68). Median baseline hemoglobin was 119 g/L (82 - 153 g/L), hematocrit 0.340 (0.250 - 0.440), and fetal hemoglobin (HbF) fraction 1% (0 - 7.7%). Most frequent complications encountered were retinopathy (55.8%) and symptomatic avascular necrosis (27.9%), followed by painful vaso-occlusive crises requiring emergency room visit (23.1%). Presence of retinopathy was associated with higher baseline hemoglobin (OR 2.72 for every 10 g/L of hemoglobin, p = 0.037) and older age (OR 2.72 for every decade, p < 0.001). Acute chest syndrome (7.7%), priapism (7.5% of men), and renal involvement (8.2%), were less common than reported in the literature, while the rates of venous thromboembolism (8.7%), symptomatic infarctive or hemorrhagic stroke (2.9%) were slightly more common. Median right ventricular systolic pressure on 2D-transthoracic echocardiogram was 29 mmHg (17 – 43 mmHg). No patient underwent a right heart catheterization. Two patients died from septic shock, both at the age of 29. Disease-modifying therapy most often consisted of hydroxyurea (28.8%), followed by exchange transfusion (6.7%) and phlebotomies (5.8%). Hydroxyurea significantly increased the median HbF fraction (from 1% to 2.75%, p = 0.004 by related-samples Wilcoxon signed rank test). Conclusion In this large North American cohort of adult patients, we have again shown that HbSC disease is not as benign as traditionally thought. As such, patients with HbSC disease warrant similar follow-up to that provided to SCA. Retinopathy, avascular necrosis and painful vaso-occlusive crises were the most common complications in our study, albeit lower than in other reported cohorts. The frequent use of hydroxyurea in this cohort is quite unique compared to other sickle cell comprehensive care centers reported in the literature. However, therapeutic phlebotomy is less often used compared to the European experience. We also observed a lower frequency of retinopathy, avascular necrosis, painful vaso-occlusive crises, priapism and acute chest syndrome. Whether this observation is due to hydroxyurea use or to other genetic or environmental factors remains to be determined. Further studies are also required to develop a more evidence-based therapeutic strategy for this genotype of Sickle Cell Disease. Disclosures: No relevant conflicts of interest to declare.

Implementation of a Pediatric Observation Unit for Children with Sickle Cell Disease

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 4838-4838
Author(s):  
Sophia Sharifali ◽  
Lashon Sturgis ◽  
Cindy Neunert ◽  
Natalie Lane ◽  
Robert Gibson ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sample Size ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Acute Chest Syndrome ◽  
Care Hospital ◽  
Cell Disease ◽  
Observation Unit ◽  
Exclusion Criteria ◽  
Direct Admission

Abstract Acute vaso-occlusive crisis (VOC), the most common manifestation of Sickle Cell Disease (SCD), is the number one cause for visits to the Emergency Department (ED). Pediatric patients differ from adult patients with SCD due to variations in opioid tolerance and age-specific complications. Many pediatric patients can be sent home after evaluation and treatment in an ED, however, others will need hospitalization for further pain management as well as continued evaluation. Observation Units (OUs), ED-associated units for evaluation and protocol management of short-term conditions (<24 hours), have successfully provided more rapid care while still maintaining quality. At our institution, using an OU-based protocol, we have demonstrated improved care with decreased resource utilization in an adult population with SCD experiencing VOC. However, there is limited data for the use of OU in pediatric patients with VOC. Our objective was to determine the feasibility of a pediatric OU for the evaluation and treatment of patients with uncomplicated VOC. A retrospective, descriptive chart review study was conducted on all pediatric patients (<18 years) with SCD between July 1, 2012 to June 30, 2013. The study was conducted in an academic pediatric tertiary care hospital (annual volume 27k/year). A medical record search was conducted using ICD-9 codes and SCD related DRG codes. The cohort was then limited to patients who received care in the academic ED or were transferred from another hospital for direct admission (DA). The cohort was limited to visits with pain related to VOC. Patients with a complication other than VOC were excluded as well as patients admitted to the intensive care unit. Cohort data as well as exclusion criteria are in table 1. Visits that were admitted to the floor (either as a direct admission or admitted from the ED) with a length of stay (LOS) less than 48 hours were included in the analysis. Patients were grouped into categories based on LOS: < 24 hours, <36 hours, and <48 hours Though the OU will only manage up to 24 hours, categories of LOS longer than 24 hours were included in order to capture elements that may lengthen a patients stay such as waiting time, time until disposition and discharge. Table 1. Sample Size and Exclusion Criteria # of Patients treated for Sickle Cell Between 7/1/2012 - 6/30,2012 197 patients Limiting to patients seen in ED or having a DA 119 patients Limiting to confirmed diagnosis of SCD (multiple genotypes) = 6 113 patients Limiting to reason of visit to a pain complaint = 6 107 patients Limiting to reason of pain to VOC = 3 104 patients Limited or no data in EMR (left prior to treatment) = 3 101 patients Exclusion of patients with visits only for complications of SCD* = 21 80 patients Final Sample Size for analysis 80 patients *Complications include acute chest syndrome, sepsis, splenic sequestration, fever, infiltrates, and infection 80 patients had 160 visits for uncomplicated VOC from 7/1/2012 - 6/30/2012. Of the 160 visits, the patient was admitted53.8% (86) of the time. Of the 86 visits resulting in admission, 30 (34.9%) were DA and 56 (65.1%) were admitted from the academic ED. LOS of the admission by DA or from the academic ED is in table 2. Table 2. LOS for Admissions DA to Floor 30 total visits LOS < 24 Hours 5 (16.6%) visits LOS < 36 Hours 10 (33.3%) visits LOS < 48 Hours 17 (56.7%) visits ED to Floor 56 total visits LOS < 24 Hours 4 (7.1%) visits LOS < 36 Hours 10 (17.9%) visits LOS < 48 Hours 21 (37.5%) visits OU's are ideal for the evaluation and management of patients requiring more than a few hours of ED treatment but less than 24 hours of hospital therapy. Our study shows that there is a large number of patients with SCD and VOC are admitted (53.8%). Based on our study, 44% of admissions have a LOS less than 48 hours. We believe that 48 hours is a reasonable cutoff for consideration of OU care as disposition decisions on the floor occur at 12-hour and sometimes 24-hour intervals leading to an increase in LOS beyond the actual treatment time. All patients, including DA patients, should be eligible for OU treatment if they meet inclusion criteria. This is evidenced by the finding that the LOS is shorter for DA patients (56.7%) versus admissions from the academic ED (37.5%). Overall, pediatric SCD patients would benefit from the presence of a pediatric OU by potentially decreasing the rate of inpatient admissions. An observation unit should therefore be strongly considered in centers with large volume SCD. Disclosures No relevant conflicts of interest to declare.

Incidence and Risk of Delayed Hemolytic Transfusion Reaction in Sickle Cell Disease Patients Based on a Prospective Study

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 95-95 ◽  
Author(s):  
France Pirenne ◽  
David Narbey ◽  
Philippe Chadebech ◽  
Armand Mekontso-Dessap ◽  
Pablo Bartolucci ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Prospective Study ◽  
Sickle Cell ◽  
Acute Chest Syndrome ◽  
Transfusion Reaction ◽  
Cell Disease ◽  
Hemolytic Transfusion Reaction ◽  
Delayed Hemolytic Transfusion Reaction ◽  
Prospective Longitudinal

Abstract Background Delayed hemolytic transfusion reaction (DHTR) is a life threatening complication of transfusion in sickle cell disease (SCD). This syndrome is underestimated because of a clinical picture that resembles a vaso-occlusive crisis (VOC) and the frequent absence of detectable antibodies. Several retrospective studies have evaluated the frequency of DHTR based on case reports. We conducted a prospective, longitudinal, single center study to determine the incidence of DHTR and the risk of developing DHTR depending on the transfusion regimen: chronic versus punctual. Methods and patients SCD patients aged over 18 years, undergoing a transfusion, were enrolled in this study. A total of 697 transfusion episodes (TE) in 312 patients were included during 30 months. Some patients had multiple TE during the period. The post transfusion outcome of the patients was assessed up to one month after the included TE. DHTR was confirmed based on the rapid disappearance of HbA (> 50% 15 days post-transfusion) associated with two of the following criteria up to three weeks after transfusion: VOC symptoms, dark urine, worsening anemia, increased LDH. Transfusion episodes were divided into chronic (336 TE in 111 patients) and punctual (361 TE in 201 patients). Chronic transfusions were defined as regular transfusions to treat chronic complications or for primary/secondary prevention of complications. Short transfusion program during pregnancy was considered as punctual transfusions if patients were not previously regularly transfused. The study obtained approval of the local Ethics Committee. Results Follow-up of the patients after transfusion showed 15 DHTR during the study. They all developed in punctually transfused patients. Thus, patients who are transfused punctually have a significantly higher risk of developing DHTR than those in a regular transfusion program (p < 0.001). When considering only punctual transfusions, the incidence of DHTR is 4.2% per transfusion episode (IC 95% [2.6; 6.9]) and 7.5% per patient during the 30 months of the study (IC 95% [4.6; 12.4]). In these DHTR cases, the transfusion indication was surgery (n = 6), pregnancy (n = 3), acute chest syndrome (n = 3), stroke (n = 1), profound anemia (n = 1), and VOC prevention before a school exam (n = 1 case). Two patients died of multi-organ failure following severe intravascular hemolysis. The median hemoglobin decrease for all DHTR cases after the triggering transfusion was 4.4 g/dl [IQR 3.6-5.2]; the highest LDH level was 879 [IQR 680-1423]. Ten patients developed newly formed antibodies, but only five among them displayed antibodies with significant serological features (anti-Fya, anti-S, anti-Jka, anti-HI). In the five other cases, the antibodies were of unspecified specificity or auto antibodies in the RH blood group (the genetic RH background was known). Finally, antibodies were undetectable for five cases, confirmed by long-term patient follow up. Conclusion This prospective study demonstrates, for the first time, that DHTR is a frequent reaction in adult SCD patients, developing only in occasionally transfused patients. This finding highlights that adult patients with regular transfusion who did not previously encountered DHTR are not susceptible to developing this severe reaction. A mechanism linked to acute situations can be suggested as already shown for the induction of allo-immunization. However, many cases developed without detectable antibodies, confirming the complex pathophysiology of this syndrome. A bio-clinical scoring system to predict DHTR, based on this study, is under development and will be presented. Disclosures Michel: Roche: Research Funding.

scholarly journals Effectiveness of surgical revascularization for stroke prevention in pediatric patients with sickle cell disease and moyamoya syndrome

2017 ◽  
Vol 20 (3) ◽  
pp. 232-238 ◽  
Author(s):  
Wuyang Yang ◽  
Risheng Xu ◽  
Jose L. Porras ◽  
Clifford M. Takemoto ◽  
Syed Khalid ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Stroke Prevention ◽  
Pediatric Patients ◽  
Moyamoya Syndrome ◽  
Cell Disease ◽  
Surgical Revascularization ◽  
Transfusion Therapy ◽  
Indirect Revascularization

OBJECTIVESickle cell disease (SCD) in combination with moyamoya syndrome (MMS) represents a rare complication of SCD, with potentially devastating neurological outcomes. The effectiveness of surgical revascularization in this patient population is currently unclear. The authors’ aim was to determine the effectiveness of surgical intervention in their series of SCD-MMS patients by comparing stroke recurrence in those undergoing revascularization and those undergoing conservative transfusion therapy.METHODSThe authors performed a retrospective chart review of patients with MMS who were seen at the Johns Hopkins Medical Institution between 1990 and 2013. Pediatric patients (age < 18 years) with confirmed diagnoses of SCD and MMS were included. Intracranial stroke occurrence during the follow-up period was compared between surgically and conservatively managed patients.RESULTSA total of 15 pediatric SCD-MMS patients (28 affected hemispheres) were included in this study, and all were African American. Seven patients (12 hemispheres) were treated with indirect surgical revascularization. The average age at MMS diagnosis was 9.0 ± 4.0 years, and 9 patients (60.0%) were female. Fourteen patients (93.3%) had strokes before diagnosis of MMS, with an average age at first stroke of 6.6 ± 3.9 years. During an average follow-up period of 11.6 years, 4 patients in the conservative treatment group experienced strokes in 5 hemispheres, whereas no patient undergoing the revascularization procedure had any strokes at follow-up (p = 0.029). Three patients experienced immediate postoperative transient ischemic attacks, but all recovered without subsequent strokes.CONCLUSIONSIndirect revascularization is suggested as a safe and effective alternative to the best medical therapy alone in patients with SCD-MMS. High-risk patients managed on a regimen of chronic transfusion should be considered for indirect revascularization to maximize the effect of stroke prevention.

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