scholarly journals Knowledge of Fertility and Perception of Fertility Treatment Among Adults with Sickle Cell Disease (KNOW FERTILITY)

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3101-3101
Author(s):  
Bria K Carrithers ◽  
Joacy Mathias ◽  
Manuela Plazas Montana ◽  
Jaanvi Mahesh ◽  
Sarah Hussain ◽  
...  
Keyword(s):  
Educational Attainment ◽  
Sickle Cell ◽  
Research Funding ◽  
Fertility Treatment ◽  
Cell Disease ◽  
Sexually Transmitted ◽  
Fertility Treatments ◽  
The Mean ◽  
Higher Educational Attainment ◽  
Perception Survey

Abstract Background Adults with sickle cell disease (SCD) face unique fertility risks due to SCD and use of disease modifying therapies (DMTs). Concerns about compromising fertility may inform patients' therapy choices, but little is known about fertility knowledge in adults with SCD. The Cardiff Fertility Knowledge Scale (CFKS) and Fertility Treatment Perception Survey have been studied in international and national cohorts 1,2. The purpose of this study was to administer these surveys to adults with SCD and compare responses to previously studied populations. Methods Our IRB approved this cross-sectional study of adults with SCD (≥18YO) cared for at our Sickle Cell Center for Adults. Due to the COVID-19 pandemic, eligible subjects were recruited during routine telemedicine clinic visits and by invitation via electronic medical record. We collected demographic information (sex, age (≥/< 31YO), educational attainment, and use of DMTs). The CFKS is a 13-question survey that measures knowledge of causes of reduced fertility, common misconceptions about fertility, and infertility facts. Questions are answered True/False/Don't know and equally weighted; the cumulative score is 0-100%. We compared the mean CFKS scores to the scores from two published cohorts 1,2. The fertility treatment perception survey consists of two positive and four negative statements about fertility treatment with responses given on a five-point Likert scale (1= strongly disagree to 5= strongly agree). Responses are calculated by number of respondents with an agreement score of 4 and/or 5 divided by total number of respondents per sub-group; higher scores indicate stronger agreement. Analysis included summary statistics with means and standard deviations and independent student's T-test to compare the mean fertility knowledge scores. Results We contacted 435 subjects; 91 respondents were enrolled (21% response rate). Respondents were 77% female [median age 33 years (IQR 23, 50)]. 51% completed high school or less and 18% used one or more DMTs, with 65% taking hydroxyurea. Table 1 shows the CFKS results. The average CFKS score was 50%, lower than the international cohort (50% vs. 57%, p<0.001) and higher than a cohort of Black women in Atlanta, GA (50% vs. 38%, p<0.001). Respondents with higher educational attainment had a higher score (55% secondary education vs. 44% primary education, p=0.04). The questions most answered correctly addressed the lack of correlation between erectile function and fertility (79%) and smoking's risk to fertility in men (69%) and women (71%). The questions least answered correctly were about classifying infertility (32%) and the impact of age effect (34%), overweight effect (25%), and sexually transmitted infections' effect on fertility (36%). There was no difference in knowledge scores by age, sex, or SCD treatment. Table 2 shows fertility treatment perception survey results. Some respondents (34%) agreed that fertility treatments are safe. Almost half (46%) agreed that fertility treatments are effective. Over 60% of respondents agreed that fertility treatments are scary and/or cause emotional problems, while 48% agreed that fertility treatments may have short-term physical effects. There was no difference in responses by sex, age, or SCD treatment. Conclusion In this study, we identify that higher educational attainment in adults with SCD is associated with better fertility knowledge. All subjects had low knowledge of sexually transmitted infections, weight gain, and older age as infertility risks. Although there is concern that hydroxyurea may compromise fertility, its use was not associated with greater fertility knowledge in this study. Given concerns about fertility in the SCD community, we identify an opportunity to support patients concerned about fertility by contextualizing real or theorized SCD-specific fertility risks within a broader set of established fertility risks. References: 1. Bunting L, Tsibulsky I, Boivin J. Fertility knowledge and beliefs about fertility treatment: findings from the International Fertility Decision-making Study. Hum Reprod. 2013 Feb;28(2):385-97. doi: 10.1093/humrep/des402. Epub 2012 Nov 25. PMID: 23184181. 2. Wiltshire A, Brayboy LM, Phillips K, et al. Infertility knowledge and treatment beliefs among African American women in an urban community. Contracept Reprod Med. 2019 Sep 24;4:16. doi: 10.1186/s40834-019-0097-x. PMCID: PMC6757383. Figure 1 Figure 1. Disclosures Lanzkron: Shire: Research Funding; GBT: Research Funding; Novo Nordisk: Consultancy; CSL Behring: Research Funding; Pfizer: Current holder of individual stocks in a privately-held company; Teva: Current holder of individual stocks in a privately-held company; Novartis: Research Funding; Bluebird Bio: Consultancy; Imara: Research Funding.

scholarly journals Emergency Department Encounters, Hospitalizations and ED Reliance Among Medicaid Eligible Patients with Sickle Cell Disease in North Carolina

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2113-2113
Author(s):  
Paula Tanabe ◽  
Nancy Crego ◽  
Christian Douglas ◽  
Emily Bonnabeau ◽  
Marian Earls ◽  
...  
Keyword(s):  
North Carolina ◽  
Sickle Cell Disease ◽  
Old Age ◽  
Sickle Cell ◽  
Research Funding ◽  
Claims Data ◽  
Age Group ◽  
Cell Disease ◽  
Old Patients ◽  
The Mean

Introduction: Sickle cell disease (SCD) is a complex disease for which pain is the hallmark. Pain from vaso-occlusive episodes is the number one reason for ED visits and hospital admissions. This paper reports Medicaid claims data from NC for individuals with SCD, including: 1) ED encounters and re-encounters within 7, 14 and 30 days; 2) hospitalization and re-hospitalization within 7, 14 and 30 days; and 3) ED reliance (EDR) score. Methods: We examined Medicaid claims data from for patients with a diagnosis of SCD (ICD 9 CM codes: 282.6x, ICD 10 CM codes: D57.0x, D57.1, D57.2x, D57.4x, D57.8x) in North Carolina. Data is reported for a cohort of 2,790 patients with a diagnosis of SCD, age 1 to 65+ and enrolled at least 11 months in NC Medicaid between March 1, 2016 and February 28, 2017. ED re-encounters and re-hospitalizations within 7, 14 and 30 days were identified using the time between the date of service listed on the ED or hospital claim and the next date of service in the subsequent claim. Individual ED Reliance (EDR) score was calculated as the total number of ED encounters divided by the total ambulatory visits (outpatient + ED encounters) per enrollee, (ambulatory visits reported elsewhere). Similar to Kroner et al, an EDR of >0.33 was considered a high score. Inpatient claims were identified using a category of service code indicating hospitalization. Results: The participants in the sample (n=2790) were majority female (57.92%), lived in metropolitan areas (77.63%) and had a mean age of 23.05 years old (SD=16.06). Of the 9,075 total ED encounters, 69.86% of the total sample had an ED encounter during the 12-month study period. There was a mean of 3.25 (SD=7.38) and median of 1 (IQR = 0 - 3) ED encounters per patient for the sample. Those who were 18-30 years old had the highest mean and median ED encounters per patient (4.98, SD= 9.34 and 2, IQR 1 to 5). The 31-45 year old group had the second most, with 4.82 (SD= 11.03) total ED encounters. The percentage of the sample with an ED re-encounter within 7, 14, and 30 days was also highest among the 18-30 year old group (29.17%, 33.98% and 40.89%) followed by those 31-45 years old (23.71%, 28.49%, and 34.80%), respectively. The 31-45 age group had the second most hospitalizations/patient and re-hospitalizations. The mean EDR was highest among 18-30 year old patients (0.35) and 46.48% of this age group had an EDR of 0.33 or greater. In the 31-45 year-old age group, the mean EDR was 0.28 and 35.18% had an EDR of 0.33 or greater. The overall sample had a mean of 1.30 (SD= 2.75) hospitalizations/patient. The 18-30 year old age group also had the highest mean total hospitalizations (2.08, SD= 3.72) and mean re-hospitalizations within 7 (0.16; SD=0.77), 14 (0.41; SD=1.68), and 30 (0.82; SD=2.79) days. The 31-45 age group had the second most hospitalizations/patient and re-hospitalizations (Table 1). Conclusions: Overall, increasing age coincided with increased ED and inpatient utilization, as well as with the period of transition from pediatric to adult SCD care. Furthermore, high EDR was most prevalent in the 18-30 age group. Our study further supports the need for increased focus on acute care utilization in the 18-45 year-old age group and considerations for improved care transition interventions. Disclosures Tanabe: NIH: Research Funding; AHRQ: Research Funding. Shah:Novartis: Consultancy, Research Funding, Speakers Bureau; Alexion: Speakers Bureau; GBT: Research Funding.

scholarly journals Effects of Hydroxyurea and Renin-Angiotensin Blockade on Kidney Function in Sickle Cell Disease

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 21-22
Author(s):  
Jin Han ◽  
Andrew Srisuwananukorn ◽  
Michel Gowhari ◽  
Faiz Ahmed Hussain ◽  
Franklin Njoku ◽  
...  
Keyword(s):  
Kidney Disease ◽  
Kidney Function ◽  
Sickle Cell ◽  
Ace Inhibitors ◽  
Research Funding ◽  
Cell Disease ◽  
Advisory Committees ◽  
Average Change ◽  
The Mean ◽  
Sickle Cell Nephropathy

Kidney disease is a common complication that leads to increased morbidity and early mortality in patients with sickle cell disease (SCD). Vaso-occlusion, hyperfiltration, hypertension, and cell-free hemoglobin/heme-mediated toxicity may contribute to the pathophysiology of kidney disease. Treatments for SCD-related kidney disease have been adopted from therapies used to treat other SCD-related complications (hydroxyurea [HU]) or diabetic nephropathy (angiotensin converting enzyme [ACE]-inhibitors or angiotensin receptor blockers [ARBs]), although their effects on kidney function are not clear. We evaluated the effects of HU and ACE-inhibitors or ARBs on kidney function in a longitudinal cohort of 439 SCD patients enrolled into a prospective registry between 2011 and 2019. Patients were considered for the analysis if they had 6 months of kidney function values pre-therapy and remained on therapy for 6 months or longer. Changes in the estimated glomerular filtration rate (eGFR) and urine albumin concentration were compared prior to and after starting therapy using a mixed effects model. The effects of HU on kidney function were evaluated in 49 SCD patients. The mean age was 38 years (standard deviation [SD] 11 years), 43% were male, and 80% were Hb SS/Sβ0-thalassemia genotype. The eGFR improved from an average decline of -3.3 mL/min/1.73m2 in the 6 months prior to starting HU to an increase of +9.5 mL/min/1.73m2 during HU therapy (P = 0.0002) (Table). The average change in albuminuria also improved from an increase of +1.2 mg/g creatinine pre-HU therapy to a decline of -1.2 mg/g creatinine during HU therapy, although the difference was not statistically significant (P = 0.17). In 47 SCD patients started on ACE-inhibitors or ARBs, the mean age was 45 years (SD 11 years), 43% were male, and 87% were Hb SS/Sβ0-thalassemia genotype. During ACE-inhibitor or ARB therapy, there was no observed difference in the change in eGFR pre- versus during therapy (P = 0.9) (Table). Albuminuria improved from an average change of -1.0 mg/g creatinine pre-therapy to -1.6 mg/g creatinine during therapy (P = 0.009). Because clinical data are limited, current American Society of Hematology guidelines have conditional recommendations with low levels of certainty for the use of HU and ACE-inhibitors or ARBs to treat sickle cell nephropathy. In a longitudinal cohort of SCD patients, we demonstrate that during 6 months of therapy, there may be a benefit of HU in improving eGFR and of ACE-inhibitors or ARBs in reducing albuminuria. Larger and longer follow up studies of HU, ACE-inhibitors and ARBs as well as new targeted therapies to treat sickle cell nephropathy are urgently needed. Figure Disclosures Gordeuk: Novartis: Consultancy; Ironwood: Research Funding; CSL Behring: Consultancy, Research Funding; Global Blood Therapeutics: Consultancy, Research Funding; Imara: Research Funding. Saraf:Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees, Other: Advisory Boards, Speakers Bureau; Novartis, Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees; Pfizer, Global Blood Therapeutics, Novartis: Research Funding.

scholarly journals Plasma-Based Inflammatory Signatures in Patients with Sickle Cell Disease during Baseline Health and Acute Pain

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 25-26
Author(s):  
Lana Mucalo ◽  
Shuang Jia ◽  
Julie Panepinto ◽  
Mark F. Roethle ◽  
Martin J. Hessner ◽  
...  
Keyword(s):  
Gene Expression ◽  
Immune Response ◽  
African American ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Acute Pain ◽  
Research Funding ◽  
Cell Disease ◽  
The Mean ◽  
Baseline Health

INTRODUCTION: Pain, the most common complication of sickle cell disease (SCD), presents as both sudden acute pain and chronic daily pain. However, there is wide variability in frequency and presentation of pain despite inheritance of the same monogenic gene defect. SCD has long been recognized as a chronic inflammatory condition. The ongoing effect of repeated vaso-occlusion, ischemia-reperfusion injury and hemolysis contribute to further SCD inflammation and likely pain. Regulation of the immune response can potentially modulate the inflammatory impact on pain. The collective balance of these inflammatory mediators in union in SCD patients and how this balance may change during baseline health and acute pain is unknown. The objective of this work was to determine the balance between patients' inflammatory and immune regulatory response and examine whether this balance changes during acute pain in patients with SCD. METHODS: We conducted a cross sectional analysis involving 3 cohorts: patients with SCD who were in their baseline health state, patients with SCD who had an acute pain episode and healthy African American controls. We used a novel bioassay originally developed for use in type 1 diabetes and applied to cystic fibrosis, inflammatory bowel disease and influenza to determine the inflammatory/immune regulatory response. This response was calculated as a composite Inflammatory Index (I.I.com) from these 3 patient cohorts. Patient plasma was co-cultured with cryopreserved PBMCs from a healthy donor to induce transcription (Figure 1). We identified informative transcripts that differentiate SCD patients from healthy controls thereby defining the disease-specific plasma-induced signature and retained ones differentially expressed between patients with SCD and controls that exhibit a fold change >1.4, ANOVA p-value of <0.05 and an FDR <10%. The data were subjected to ontological analyses for quantitative interpretation with Database for Annotation, Visualization and Integrated Discovery (DAVID) and Ingenuity Pathway Analysis (IPA). Our scoring strategy used the degree of induction of genes in inflammatory and regulatory ontological classes. The composite I.I.com was calculated using the average ratio between the mean log intensity of the genes classified as being "inflammatory" versus "regulatory". Independent samples Student's t-test was used to compare the mean I.I.com between 1) SCD baseline health cohort and controls and 2) SCD baseline health cohort and SCD acute pain cohort. RESULTS: Plasma from 16 patients with SCD in baseline health, 27 patients with SCD with an acute pain episode, and 45 African American controls were collected and analyzed. The average age of the study population was 12.6 (SD=3.6) years old and 52.3% were female. Quantitative scoring of plasma-induced signatures showed SCD patients had significantly higher mean I.I.com during baseline health compared to controls (0.713 vs. -1.235-12, p=5.4625-11). In addition, patients with SCD during acute pain episodes had significantly higher I.I.com than patients in baseline health (1.282 vs. 0.713, p=5.2051-8) (Figure 2). Heat map in Figure 3 shows differential gene expression between the cohorts; green and red colors in heat maps represent lower or higher relative expression respectively. CONCLUSION: Our findings show distinct immune signatures in SCD patients compared to controls and distinct signatures in SCD patients during acute pain episodes as compared to baseline health. The novel assay used to assess the inflammatory and immune regulatory gene expression in the three cohorts studied allowed for the determination of the balance between the two immune states. The imbalance between inflammation and immune regulation shown in our results in SCD patients of SCD pain. Further investigation into the specific inflammatory pathways that contribute to altered immune response could lead to novel targets for pain treatment. Disclosures Mucalo: NIH/NINDS: Research Funding; NIH/NHLBI: Research Funding. Jia:NIH/NHBLI: Research Funding; NIH/NINDS: Research Funding. Panepinto:NINDS: Research Funding; HRSA: Research Funding; NINDS: Research Funding; NHLBI: Research Funding. Roethle:NIH/NHLBI: Research Funding; NIH/NINDS: Research Funding. Hessner:NIH/NHLBI: Research Funding; NIH/NINDS: Research Funding. Brandow:NIH / NHLBI: Research Funding; Greater Milwaukee Foundation: Research Funding.

Feritility and Attitudes To Fertility In a Cohort Of Women With Sickle Cell disease In South East London

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 4686-4686
Author(s):  
Rachel Kesse-Adu ◽  
Ronwyn Cartwright ◽  
Nicky Thomas ◽  
Eugene Oteng-ntim ◽  
Jo Howard
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Clinical Severity ◽  
Fertility Treatment ◽  
In Vitro Fertilisation ◽  
Disease Phenotype ◽  
Cell Disease ◽  
The Mean ◽  
The Impact ◽  
Age Of Menarche

Introduction Women with Sickle Cell Disease (SCD) have been reported to have delayed menarche, increased rates of miscarriage & still birth. There is little evidence of the impact of SCD severity on fertility, or of patients’ attitudes towards their fertility. Methods Following IRB approval, an anonymised questionnaire was distributed to female patients over the age of 16 attending an SCD clinic. This included questions on menarche, fertility, reproductive history & clinical severity. We also surveyed attitudes to fertility & the role of fertility treatment. Results 101 women gave informed consent & returned the survey of whom 68 (67%) had sickle cell anaemia (HbSS), 31 (31%) had HbSC & 2 (2%) HbSß0 thalassaemia (HbSß0thal), the group mean age was 38yrs (range 17-74). 46 patients had severe disease phenotype (criteria for severity: ≥3 hospital admissions per year / ≥2 episodes of acute chest crisis/ ≥6 crisis at home per year or on Hydroxycarbamide (HU) therapy), 78% (36/46) had HbSS & 49%(2/46) HbSC. 49% (22/46) were on HU, of whom 19 (86%) had HbSS & 3 (14%) HbSC, the mean duration on HU was 4.7 years (range 2 weeks–15 years). 3 patients with HbSS, were on a chronic transfusion program, 1 as secondary stroke prevention & 2 for disease severity. Mean age of menarche was 14 yrs (range 10-19). HbSC patients’ mean age of menarche was 13yrs (range 10-16), HbSS patients mean age of menarche was 14 yrs (range 10-19). 30% (30/101), (24 with HbSS & 6 with HbSC), had never been pregnant. This group had a mean age of 28yrs (range 17-46), 1 never wanted children, 2 had previously been advised not to conceive, 1 was actively trying for a baby, & 1 patient was awaiting in vitro fertilisation therapy (IVF). There was no difference in the severity of sickle disease in this group compared to the group with pregnancies. 70%(71/101) reported a total of 211 pregnancies, resulting in 111 (52%) successful deliveries (SDs) i.e. a live child/children. Further evaluation of 175 (81%) pregnancies revealed 98 (56%) SDs, (5 sets of twins & 1 early neonatal death equating to 102 live births), 45 (26%) miscarriages, 27 (15%) terminations of pregnancy & 5 (3%) still births (pregnancy loss at > 24 weeks gestation). 52% (50/96) & 63% (47/75) of pregnancies in women with HbSS & HbSC resulted in SDs respectively, as did 25%(1/4) of pregnancies in HbSß0thal women. 74% (53/71) had at least 1 miscarriage (mean 1.7, range 1-7) there was no correlation between miscarriage & sickle genotype or age at pregnancy. 46% (20/46) & 63% (36/55) of patients with severe & milder disease phenotype had miscarriages respectively (p = 0.01). The mean age at termination of pregnancy (26yrs) was lower, compared to 30yrs for women who had SDs or miscarriage & stillbirth (31yrs). In total 41% (72/175) pregnancies were unplanned. 96% (26/27) of terminated pregnancies were unplanned, as were 32% (31/98) of SDs & 28% (15/50) of miscarriages. 28% (28/100) of responders were currently using contraception. 12 (16%) of 71 women reporting pregnancies had no children, they had a total of 21 pregnancies (mean 1.75, range 1-7), 8 (67%) had HbSS, 3 (25%) HbSC. 18% (18/101) thought sickle may have affected their fertility, 93% (93/101) reported having children as being important, this included 28 (93%) of 30 women who had reported no pregnancies. 62% (63/101) would consider prenatal diagnoses (PND) if their partner had sickle cell trait, 59% (60/101) would consider pre implantation diagnosis (PIGD) & 24% ( 24/101) would consider termination of an fetus with SCD. Discussion This study confirms that compared to the normal UK population, women with SCD, have a slightly increased rate of miscarriage & a significantly elevated rate of stillbirth 3% vs 0.005%. Menarche is delayed by a year in patients with HbSS & women with HbSC are more likely to have a successful pregnancy. Although 74% of the women had miscarriage & only 56% of pregnancies resulted in a successful delivery, the majority of women were unaware of the effect of SCD on fertility. Most were not currently using any form of contraception, the rate of unplanned pregnancy was lower at 41% than that in the normal UK population of 50%. These women report having a child as being very important to them, & though majority would consider PND & PIGD only a minority (24%) would terminate a foetus diagnosed with SCD. This study is limited by being retrospective & single centred, further multi-centre prospective studies are required to validate these results. Disclosures: Howard: Sangart: Membership on an entity’s Board of Directors or advisory committees.

Episodic Patterns of High Emergency Department Utilization Among Sickle Cell Disease Patients

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 316-316
Author(s):  
Susan Paulukonis ◽  
Lisa Feuchtbaum ◽  
Elliott Vichinsky ◽  
Mary Hulihan
Keyword(s):  
Emergency Department ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Surveillance System ◽  
Research Funding ◽  
Cell Disease ◽  
Ed Visits ◽  
High Utilization ◽  
The Mean ◽  
Over Time

Abstract Background: High utilization of emergency department (ED) services among those with sickle cell disease (SCD) compared to the general population and compared to those with other chronic diseases is well documented in the literature. Some reports note that high utilization is episodic. Most analyses address the problem as a consistent one within patients, rather than consistent over time across the patient population but sporadic for patients. Reducing the high rate of ED utilization among patients with SCD requires an understanding of temporal patterns of ED utilization, the consistency of ED utilization over time by patients and the proportion of the population affected at any given time. Methods: CDC has developed the Sickle Cell Data Collection program (SCDC) to conduct state level surveillance in this disease, and to continue and improve upon work begun through the Registry and Surveillance System in Hemoglobinopathies (RuSH). Through SCDC, California has collected ED and hospitalization data for years 2005-2014 on 4,325 people with SCD. A period of high ED utilization among this cohort was defined as three or more ED encounters (either treat and release or admission to the hospital from the ED) for any diagnosis (not only SCD diagnoses) each fewer than 30 days from the prior visit. The start of an episode of high utilization is the date of the first ED encounter; the end is the date of the last eligible ED encounter. All cohort members were divided into categories of utilization using the proportion of time spent in periods of high utilization divided by the total time in cohort. Total time is cohort is defined as the length of time from the earliest appearance in the ED or hospital data 2005-2014 to the latest appearance. The five categories were defined as no episodes of high ED utilization, and approximate quartile groups for those with high ED utilization: 1.1 to 3.0%, 3.1-10.0%, 10.1% or greater. Age categories (pediatric is < 21 years, and adult is 21 years or older) are defined as patient age at close of study (end of 2014) or at death if prior. Patient ID beginning with P is a pediatric, A is adult in the figures. Results: There were 4,325 individuals with 27,694 person years in the cohort (mean 6.4 person years, median 7.6 person years). Sixty-three percent (n=2,715) of the cohort were aged 21 years and older. Forty-five percent, (n=1,955, 513 pediatric and 1,442 adults) had at least one episode of high utilization during the 10 year study for a total of 7,866 episodes of high utilization. Forty-three percent of patients with one or more high utilization episodes were male, and 63% were between the ages of 20 and 50. Nine percent of these high utilizing patients' total time in the cohort was made up of episodes of high utilization. The mean time span from start of episodes of high utilization to end of the episodes was 63.3 days, median 35 days; mean number of ED visits per episode was 9.0, median 4.0. Most episodes of high utilization were brief: 42.2% included just three visits, and 70.7% included five or fewer ED visits. Among these individuals with episodes of high utilization, the mean number of such episodes was 4.0 over the 10 years study period, and the median was 2.0. Most (76.4%) had five or fewer high utilization episodes, and 35.5% had just one (n = 693). Sample utilization patterns, including hospital admissions, are shown in Figure 1. Conclusions: We demonstrate that among individuals with SCD seen in a population-based, statewide surveillance system, periods of high ED utilization are common, but most SCD patients have only a limited number of short episodes of such utilization. We found that high ED utilization is episodic rather than consistent within individuals, and that while the range of time spent in episodes of high utilization varies, few patients are high utilizers of ED services over a long period of time. Statewide surveillance that follows individual patients over time and in different hospital settings and includes ED utilization (including visits not coded as being related to SCD), provides high quality public health information to inform clinicians and healthcare systems in their development of efforts to reduce ED utilization among those living with SCD. Figure 1 Figure 1. Disclosures Paulukonis: Pfizer: Research Funding; Biogen: Research Funding.

scholarly journals Is There a Clinical Benefit to Switch Hydroxyurea (HU) Drug in Sickle Cell Disease (SCD)?

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 4-5
Author(s):  
Frédéric Galactéros ◽  
Ersi Voskaridou ◽  
Anoosha Habibi ◽  
Giovanna Cannas ◽  
Laure Joseph ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Board Of Directors ◽  
Sickle Cell ◽  
Clinical Benefit ◽  
Research Funding ◽  
Fine Tuning ◽  
Cell Disease ◽  
Advisory Committees ◽  
Previously Treated ◽  
The Mean

The European Sickle Cell Disease Cohort - Hydroxyurea (ESCORT-HU) study was initiated when HU got an approval in SCD in Europe. This non-interventional prospective cohort study conducted in patients treated with HU according to current clinical practice with SCD was terminated in 2019 after enrolment of 1906 patients in 4 European countries. The main objective was to refine the safety profile of hydroxycarbamide, as well as to identify unexpected toxicities, especially after long-term treatment. However clinical effectiveness evaluated by recording painful crises lasting more than 48 hours, episodes of ACS, number of hospitalisations related to SCD, and biological parameters were also regularly recorded according to the frequency of the hospital visits. At inclusion, 926 (48.7%) patients had been previously treated off the label with HU (as HU was only approved for myeloproliferative disorders at the time), with mean duration of HU treatment of 5.74 ± 4.98 years. The mean age of this subgroup was 27.60 ± 14.81 years compared to 19.75 ± 15.79 years of the patients never been treated with HU before enrolment Of the 926 patients, 299 (32%) were younger than 18 years. As showed in the table 1, hemoglobin level remains unchanged during the first two years but the HbF% increased in the HU-pretreated subgroup. In the "HU-naïve group", despite lower baseline Hb level et HbF% at baseline , similar outcome were obtained after 12 or 24 months. In terms of clinical outcomes, after 1 year of treatment the number of vaso-occlusive crises (VOC), acute chest syndrome (ACS) or hospitalizations decreased dramatically (table 2) in both group. The median duration of follow-up in the cohort was 45 months (0-128). 123.7 ± 62.7 months was the mean total exposure to HU in the HU-pretreated subgroup. In term of safety, neutropenia, thrombocytopenia and dry skin were the most frequent HU- related adverse events reported but with comparable cumulated incidence between the HU-pretreated subgroup and HU-naïve subgroup (Table 3). Conclusion: In real life setting, a significant improvement of the vasoocclusive symptoms was observed. Improvement of the compliance thanks to a treatment dedicated to the disease is probably one reason for better effectiveness; as suggested bythe lower red blood cell MCV was lower than expected at baseline in patients previously treated with HU. It is also possible that the increase in HbF% observed during the treatment could be another reason for clinical benefit. Paule and al (2011) demonstrated that daily regimen of HU were superior to weekly regimen. The fine tuning of the daily dose possible with HU tablets might be another reason of clinical optimization. Disclosures Galactéros: Addmedica:Membership on an entity's Board of Directors or advisory committees.Voskaridou:BMS:Consultancy, Research Funding;ADDMEDICA Company:Consultancy, Research Funding;NOVARTIS Company:Research Funding;GENESIS Company:Consultancy, Research Funding;PROTAGONIST Company:Research Funding;ACCELERON Company:Consultancy, Research Funding.Habibi:Pfizer:Consultancy;Bluebird:Consultancy;Novartis:Consultancy;Addmedica:Consultancy.De Montalembert:bluebird bio:Honoraria, Membership on an entity's Board of Directors or advisory committees;Vertex:Honoraria, Membership on an entity's Board of Directors or advisory committees;Addmedica:Honoraria, Membership on an entity's Board of Directors or advisory committees;Novartis:Honoraria, Membership on an entity's Board of Directors or advisory committees.

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.

scholarly journals Outcomes of Pregnancies in Patients with Sickle-Cell Disease : Update from European Non-Interventional, Multicentric, Prospective Escort-HU Study

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 891-891
Author(s):  
Frédéric Galactéros ◽  
Giovanna Cannas ◽  
Pablo Bartolucci ◽  
Ersi Voskaridou ◽  
Laure Joseph ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Board Of Directors ◽  
Sickle Cell ◽  
Live Birth ◽  
Research Funding ◽  
Current Practice ◽  
Cell Disease ◽  
Advisory Committees ◽  
Live Births ◽  
The Mean

Hydroxyurea (HU) is approved in EU and USA for the prevention of vaso-occlusive crises (VOC) including acute chest syndromes (ACS) in patients over 2 years with sickle-cell disease (SCD). Patients on HU wishing to conceive should stop treatment 3 to 6 months before pregnancy if possible. Pregnancy in SCD female patients are considered at risk for the mother and the fetus. This condition is associated with increased pain, infections, thromboembolic events,[1] and vaso-occlusion in placenta can lead to adverse fetal outcomes.[2] A few cases of HU exposure during pregnancy in SCD patients previously published[3],[4] suggested that the risk of deleterious teratogenic effect of HU shown in animal species[5] may have been overestimated in humans.[6] Therefore, a much larger dataset of pregnancies with HU exposure was needed. ESCORT-HU study (European Sickle Cell Disease COhoRT - HydroxyUrea) is a multicentric, prospective, non-interventional European study initiated to collect information about long-term safety of HU when used in current practice. This is the first study in which all pregnancy courses were collected irrespectively of action taken with HU before or during pregnancy. Overall 1906 patients were enrolled from 63 centers in France, Germany, Greece and Italy, 854 men (45%) and 1052 women (55%) among them around 2/3 aged from 15 to 49 years during the follow-up. During the study, 125 pregnancies in 101 women and 12 pregnancies in 10 partners of male patients were collected in the study, regardless HU exposure. In pregnancies with HU paternal exposure, 10 live births and 2 miscarriages were reported. Durations of HU exposure and outcomes of pregnancies in females treated with HU are provided in Table 1. The mean age at the pregnancy was 30 years. The mean HU duration before pregnancy was nearly 5 years. In only 16 pregnancies with maternal exposure (15%) HU was stopped at least 15 days before conception. In 43 pregnancies (34%), transfusions were reported. Live births were reported in 73% of pregnancies with maternal HU exposure excluding voluntary abortions. There was no statistical difference (Chi-squared test) in the proportions of live birth in exposed and non-exposed females (p=0.577). VOC or ACS have been reported in 3 women after the stop of HU. Although women are advised to stop HU before pregnancy, in current practice continuation of HU may be required and HU remains the only alternative to protect the mother and the fetus from deleterious effects of VOC. In conclusion, the data on pregnancy outcome following HU exposure are reassuring when compared to those in general SCD population. Overall in ESCORT-HU, 61% of pregnancies resulted in live birth, which is more than the 42% of pregnancies in the MSH study participants.4 It is notable, without considering voluntary abortions, that the percentage of live birth in ESCORT-HU reached 71%. The rate of preterm delivery (13%) was similar to the one (16%) in HbSS patients from a French cohort.[7] The fertility in women treated with HU seems to be even better and no particular problem in newborns has been reported to date. Disclosures Galactéros: Addmedica: Membership on an entity's Board of Directors or advisory committees. Cannas:Addmedica: Membership on an entity's Board of Directors or advisory committees. Bartolucci:AddMedica: Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Membership on an entity's Board of Directors or advisory committees; Agios: Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees; Roche: Membership on an entity's Board of Directors or advisory committees; HEMANEXT: Membership on an entity's Board of Directors or advisory committees. Voskaridou:Addmedica: Membership on an entity's Board of Directors or advisory committees; Acceleron: Consultancy, Research Funding; Genesis: Consultancy, Research Funding; Protagonist: Research Funding; Celgene Corporation: Consultancy, Research Funding.

scholarly journals Five-Year Outcomes of Chronic Red Blood Cell Exchange Transfusion Program for Patients with Sickle Cell Disease, the Experience of University of Nebraska Medical Center

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4988-4988
Author(s):  
Omar Abughanimeh ◽  
Steven Ebers ◽  
Mahammed Khan Suheb ◽  
Julie Eclov ◽  
Robin High ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Blood Cell ◽  
Emergency Room ◽  
Sickle Cell ◽  
Red Blood Cell ◽  
Research Funding ◽  
Medical Center ◽  
Emergency Room Visits ◽  
Cell Disease ◽  
The Mean

Abstract Background: Red blood cell exchange (RBCX) is an effective therapy in treatment of acute and chronic complications of sickle cell disease (SCD). It involves exchanging patient's red blood cells (RBCs) with donor RBCs to significantly lower hemoglobin S concentration without subjecting the patient to the risk of iron overload. The University of Nebraska Medical Center (UNMC) established a chronic RBCX program in November 2015, which cared for patients with multiple hemoglobinopathies. In this study, we aim to evaluate some of the outcomes of patients with SCD who joined the program. Methods: This is a retrospective study based on review of medical records of patients with sickle cell disease. We reviewed the health records of patients with SCD who were enrolled in the chronic RBCX program between 11/2015-8/2020 at UNMC. We included patients with SCD, regardless of age, who underwent RBCX in the outpatient setting during the study period. Data were collected to assess if RBCX influenced the frequency of SCD crisis, emergency room visits, hospitalizations, and other sickle cell-related complications. Results: A total of 404 sessions of exchange transfusions were performed between November 2015 and August 2020 for 21 patients with SCD. The study included 9 adults (age ≥ 18 years) and 12 children with a median age of 12 years (2-31 years). During the study period, 3 adults left the program due to relocation out of state, patient's preference, or physician's decision. Table 1 summarizes the population demographic. The most common indication for enrollment in the RBCX program was recurrent sickle cell crisis (Figure 1). The mean number of emergency room visits before enrollment in the RBCX program was 22.5 visits (2-62 visits), which reduced after enrollment to 10.4 visits (0-65 visits), with a difference in mean of 12.1 visits (P=0.0021). The mean number of hospital admissions before enrollment in the RBCX program was 13.2 admissions(0-54 admissions), which also reduced to 6.7 admissions (0-50 admissions), with a significant difference in the means equal to 6. 6 admissions (P=0.0013) (Figure 2). Thirteen patients had a baseline ferritin &gt; 500 ng/ml at enrollment; all of them had a decrease in their baseline ferritin during the study, with 4 of them achieving a new baseline &lt; 500 ng/ml. Six patients had pre-existing antibodies at enrollment due to prior alloimmunization; however, no new alloantibodies were noticed after enrollment. The patients without preexisting antibodies were transfused with Rh and Kell matched blood. The patients with pre-existing antibodies were transfused with phenotypically matched blood. Three patients became pregnant during the study period, and their pregnancies were uncomplicated except for one patient with preeclampsia resulting in early delivery. There was no reportable death, acute chest syndrome, or stroke among the patients during the study period. Conclusion Outpatient chronic RBCX demonstrated safety and feasibility in both adults and children. It also showed promising outcomes in terms of reduction of sickle cell complications, number of emergency room visits and hospitalizations. These results can provide the basis for evaluating RBCX in a prospective study to better understand changes in quality of life and clinical outcomes of patients with SCD and limited therapeutic options. Figure 1 Figure 1. Disclosures Gundabolu: Pfizer: Research Funding; Samus Therapeutics: Research Funding; BioMarin Pharmaceuticals: Consultancy; Bristol-Myers Squibb Company: Consultancy; Blueprint Medicines: Consultancy.

scholarly journals Prevalence and Predisposing Factors of Atrial Fibrillation in a Multi-Ethnic Society: The Impact of Racial Differences in Bahrain

2011 ◽  
Vol 4 ◽  
pp. OJCS.S8032 ◽  
Author(s):  
Taysir Garadah ◽  
Saleh Gabani ◽  
Mohamed Al Alawi ◽  
Ahmed Abu-Taleb
Keyword(s):  
Atrial Fibrillation ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Middle Eastern ◽  
Predisposing Factors ◽  
Cell Disease ◽  
Clinical Events ◽  
History Of ◽  
The Mean ◽  
One Year

Background The prevalence and epidemiological data of atrial fibrillation (AF) among multi-ethnic populations is less well studied worldwide. Aim Evaluation of the prevalence and predisposing factors of AF in patients who were admitted to acute medical emergencies (ER) in Bahrain over the period of one year. Methods Two hundred and fifty three patients with onset of AF were studied. The mean difference of biochemical data and clinical characteristics between Middle Eastern (ME) and sub continental (SC) patients was evaluated. The odds ratio of different predisposing factors for the development of clinical events in AF patients was assessed using multiple logistic regression analysis. Results Out of 7,450 patients that were admitted to ER over one year, 253 had AF based on twelve leads Electrocardiogram (ECG), with prevalence of 3.4%. In the whole study, the mean age was 59.45 ± 18.27 years, with 164 (65%) male. There were 150 ME patients (59%), and 107 (41%) SC, 55 (22%) were Indian (IND) and 48 (19%) were South Asian (SA). In the whole study clinical presentation was of 48% for palpitation, pulmonary edema was of 14%, angina pectoris on rest of 12%, 10% had embolic phenomena, 6% had dizziness, and 7% were asymptomatic. The odds ratio of different variables for occurrence of clinical events in the study was positive of 2.2 for history of hypertension, 1.8 for sickle cell disease, 1.2 for high body mass index (BMI) >30, 1.1 for mitral valve disease. The ME patients, compared with SC, were older, had significantly higher body mass index, higher history of rheumatic valve disease, sickle cell disease with high level of uric acid and lower hemoglobin. The history of hypertension, DM and smoking was higher among the SC patients. The rate of thyroid disease was equal in both groups. Conclusion The prevalence of atrial fibrillation was 3.4% with male predominance of 65%. Patients of sub continental origin were younger with a significantly high history of hypertension and ischemic heart disease. The patients of Middle Eastern origin had significantly high rate of rheumatic heart disease, and sickle cell disease. The history of hypertension was the most important independent clinical predictor of adverse events in patients presented with AF.

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