scholarly journals Transfusion Therapy in a Multi-Ethnic Sickle Cell Population Real-World Practice. a Preliminary Data Analysis of Multicentre Survey

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 2389-2389 ◽  
Author(s):  
Giovanna Graziadei ◽  
Laura Sainati ◽  
Pietro Bonomo ◽  
Donatella Venturelli ◽  
Nicoletta Masera ◽  
...  
Keyword(s):  
Sickle Cell ◽  
Research Funding ◽  
Acute Chest Syndrome ◽  
Therapeutic Approaches ◽  
Transfusion Therapy ◽  
Acute Anemia ◽  
Italian Regions ◽  
Number Of Patients ◽  
Significant Difference ◽  
Caucasian Patients

Abstract Introduction. Despite the increasing of number of patients with Sickle Cell Disease (SCD) in Italy, due to multi-ethnic migratory phenomena, a large percentage of Caucasian sickle population is already present in Italy mainly with b-thal/HbS genotype. Red cell transfusion is one effective treatment for both acute and chronic complications of SCD, while hydroxycarbamide (HC) is used to reduce the frequency of painful vaso-occlusive crises (VOCs) and decrease the need for blood transfusion. Through the National Comprehensive Reference Centers for SCD, the Italian Society of Thalassemia and Hemoglobinopathies (SITE), in collaboration with the Society Italian Transfusion Medicine and Immunohematology (SIMTI) and the Italian Association of Hematology and Pediatric Oncology (AIEOP) conducted a national survey to collect information on different therapeutic approaches used for SCD patients. Aim. To assess therapeutic approaches used a large Italian cohort of patients with SCD, accounting for age, genotype and ethnicity. Patients and Methods. Observational Longitudinal Systemic Multicentre Study (https://clinicaltrials.gov/ct2/show/NCT03397017). Data were collected from 2015 to 2018 through a standard web-based application (www.SITE-italia.org) encrypted by the Central Server. All the SCD patients, treated or not treated, were included in order to identify the overall number and all gave written informed consent. The study was approved by Ethics Committee of Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico of Milan, Italy. Results. Thirty-four centers were involved from 14 Italian regions and 1,579 patients were enrolled (802 male and 777 female; median age 23 years - IQR, 25th-75th 10-41 yrs). Genotype, age and ethnicity distribution are shown in Table 1A. As expected, the median age of non-Caucasian patients, mainly HbSS, is significantly lower than Caucasian ones (p<0.001). Out of 1,579, 365 SCD patients (23%) did not receive any therapy. Acute transfusion regimen (ATR), Chronic transfusion regimen (CTR) and HC were given in monotherapy, respectively in 160, 226 and 197 patients, or in succession/combination in 631 (Table 1B), distributed throughout genotypes. The main reasons for ATR were acute anemia (384 events) and VOCs (352), followed by acute chest syndrome (ACS; 170), surgery (82), pregnancy (64), splenic sequestration (26), stroke (9); multi-organ failure (MOFs 6) and priapism (5). For CRT, it was acute anemia (306 events) and prevention of VOCs (371), ACS (107), primary stroke prevention (78) and secondary prevention stroke (55), pain HC-resistent (39) and leg ulcers (12). For 275 patients out of 631 it was possible to follow the timing of therapy switching (Table 1C). Of 275 patients, 67.6% switched from ATR/CRT to HC, 2.9% from HC to CRT and 6.5% stopped every therapy. Out of 275 patients, 104 were treated with overlapping therapeutic regimen. Discussion. The significant difference of age in Caucasian and non-Caucasian patients is probably due to the efficacy of the national prevention program of hemoglobinopathies, because the non-Caucasian patients are prevalently born out of Italy. The transfusional approach is similar in HbSS and b°-thal/HbS and b+-thal/HbS patients regarding both ATR and CTR. HbSC genotype needed less therapies(p <0.001), confirming a less severe clinical pattern. About the combo or sequential therapy, HC was the more frequent chronic therapy used lifelong, mainly in patients with HbSS, because of wide spread of age and transfusional match problems due to different ethnicity. Summary/Conclusion. The transfusional approach is similar in HbSS, b°-thal/HbS and b+-thal/HbS patients with similar indications, prevalently VOCs and anemia. The significant higher age in Caucasian cohort and the consequent long term follow up could be the cause of variable therapeutic approach observed, however Hydroxycarbamide seemed to be the therapy more frequently used and finally suggested to manage chronic manifestations. Figure. Figure. Disclosures Origa: Apopharma: Honoraria; Novartis: Honoraria; Bluebird Bio: Consultancy; Cerus Corporation: Research Funding. Forni:Apopharma: Other: DSM Board; Celgene: Research Funding; Novartis: Other: travel expenses, Research Funding; Shire: Research Funding; Roche: Consultancy.

Clinical Complications in Adult Patients with Sickle Cell Anemia and β-Thalassemia-Sickle Cell Disease

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4767-4767
Author(s):  
Giovanna Graziadei ◽  
Alessia Marcon ◽  
Martina Soldarini ◽  
Ilaria Gandolfi ◽  
Luisa Ronzoni ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Acute Chest Syndrome ◽  
P Value ◽  
Cell Disease ◽  
Transfusion Therapy ◽  
Clinical Complications ◽  
Significant Difference ◽  
The Mean

Abstract Abstract 4767 Background. Sickle-Cell Disease (SCD) is one of the most common severe monogenic inherited disorders worldwide, due to hemoglobin S (HbS), with reduced affinity for the oxygen. HbS polymerization, leading to erythrocyte rigidity, vaso-occlusion and hemolytic anemia, is central in the pathophysiology and crucial for the clinical outcome. The term SCD refers to Sickle Cell Anemia (SCA) due to homozygosis for βS allele, HbS/β-thalassemia (T-SCD) due to compound of β-thal and βS allele, and HbSC disease, owing to the coinheritance of βS and βcalleles. SCD is a multiorgan disease characterized by recurrent acute events and progressive organ damage, worsening during the life. Aims. This is a retrospective monocentric study aimed to assess and compare the clinical complications among 59 adult SCD patients, followed at the Hereditary Anemia Centre of the Foundation IRCCS “Ca Granda” Ospedale Maggiore Policlinico, in Milan, Italy. Methods. Mutation analysis of the b globin gene was established by direct DNA sequencing on the ABI Prism 310 genetic analyzer. Clinical and hematological features were evaluated by routine tests and physical examination, with special attention to the erythropoiesis stress parameters as LDH values and extramedullary erythropoietic (EE) masses. Results. Fifty-nine adult SCD patients, 16 SCA and 43 T-SCD, were evaluated. In T-SCD patients detected b-mutations were severe (b°) in 69.8%, and moderate or mild (b+-b++) in 30.2%. The mean age of SCA patients was 36±9 and 41±11 years for T-SCD patients. For both groups the mean follow-up was 20±6 years, while the mean age at the presentation in our Centre was 32±8 years in SCA patients and 31±10 years in T-SCD ones. Five out of 16 (31.2%) SCA patients and 16/43 (37.2%) T-SCD patients were male. HbF mean levels were 6.9±5.1% and 10.1±7.2%, respectively in SCA and T-SCD group; surprisingly Hb mean levels were lower in SCA (9.3±1.3 g/dl) than in T-SCD (9.9±1.4 g/dl) patients. Comparing SCA and T-SCD, there was statistically significant difference in splenic features: splenectomy was performed in 2/16 (12.5%) SCA patients vs 21/43 (48.8%) T-SCD patients (p-value < 0.01). Splenomegaly was absent in SCA, while was detected in 11/22 (50%) T-SCD (p-value < 0.0001); all SCA patients had functional asplenia, not observed in T-SCD patients; splenic infarctions were absent in SCA patients and were detected in 7/22 (31.8%) T-SCD patients, of whom 5 had splenomegaly and 2 had normal spleen size (pvalue <0.001). On the other side, there was not statistically significant difference in the prevalence of stroke, acute chest syndrome (ACS), bone pain crisis, sepsis, leg ulcers and priapism. However, we observed some clinical differences, even if not statistically significant. Cholecistectomy was performed in 4/16 (25%) SCA patients vs 17/43 (39.5%) T-SCD patients, and gallstones were detected respectively in 5/12 (41.7%) and in 14/26 (53.8%) of SCA and T-SCD patients. Thrombotic events were absent in SCA patients, compared to 4/43 (9.3%) T-SCD patients. Furthermore, we detected EE in 3/16 (18.6%) SCA and in 3/43 (7%) T-SCD, all carrying b° thal mutations. We underlie that Hb levels and LDH values were higher in SCA than in T-SCD patients (823±295 vs 689±209 U/L). About the treatment, 14/16 (87.5%) SCA and 31/43 (72%) T-SCD underwent to top-up transfusion; 5/43 (11.6%) T-SCD were regularly transfused. Seven out of 16 (43.8%) SCA and 18/43 (41.8%) T-SCD patients were treated with Hydroxycarbamide (HU). Criteria for transfusion therapy were: painful crisis not responsive to HU, major clinical complications, such as stroke or ACS, extramedullary erythropoietic masses associated with high LDH levels and low Hb values. Conclusions. These data suggest that SCA and T-SCD patients have similar clinical course. Splenomegaly is present only in T-SCD patients, probably due to the increased amount of extravascular hemolysis. Surprisingly, SCA patients showed EE and lower Hb levels with higher LDH values compared to T-SCD ones. This could be related to the prevalence of intravascular hemolysis, that can lead to erythropoietic stress in SCA, even if tissues are better oxygenated in these patients because of biochemical characteristic of HbS in terms of decreased oxygen affinity. These observations could be important to evaluate transfusion and HU treatment. Disclosures: Cappellini: Novartis: Research Funding.

Impact of Hydroxyurea On Peri-Operative Management and Outcomes in Children with Sickle Cell Anemia.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 2567-2567
Author(s):  
Masanori Hayashi ◽  
Agustin Calatroni ◽  
Brittany Herzberg ◽  
Courtney Thornburg
Keyword(s):  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Operative Management ◽  
Wilcoxon Test ◽  
Categorical Variables ◽  
Acute Chest Syndrome ◽  
Transfusion Therapy ◽  
Significant Difference ◽  
Operative Complications ◽  
Post Operative Complications

Abstract Abstract 2567 Poster Board II-544 Surgical procedures in children with sickle cell anemia (SCA) can be complicated by vasoocclusive events (VOE) such as acute chest syndrome (ACS) and pain. Peri-operative management requires a multidisciplinary approach to provide appropriate pre-operative intravenous hydration and intra- and post-operative monitoring. Transfusion therapy has been controversial. Our institution previously described a low incidence of complications in children who received serial transfusions over 3-4 weeks prior to surgery. Subsequently, an increasing number of children have been prescribed hydroxyurea (HU) to prevent SCA complications. In general, children on HU at our institution only receive a single top-off transfusion the day prior to surgery if their hemoglobin is less than 10 g/dL. We hypothesized that children in the HU group would have a lower number of serial transfusion compared to the non-HU group and that there would be no difference in complications or days to discharge between the two groups. We conducted a single-institution retrospective cohort study of children with SCA, who were age less than 18 years and underwent at least one surgical procedure at Duke University Medical Center between January 1, 2003 and April 30, 2008. Data were abstracted from electronic and written medical records. Descriptive statistics were used to characterize the cohort. Wilcoxon test was used to compare continuous variables and Pearson test was used to compare categorical variables between the non-HU and HU groups. Fifty-three subjects were included (Table 1). The non-HU group was significantly younger than the HU group, but children in the non-HU group were significantly more likely to be transfused pre-operatively, primarily with serial transfusions or erythrocytopheresis, compared to the HU group. One subject in the non-HU group developed a pre-operative delayed hyperhemolytic transfusion reaction. Post-operative complications are detailed in Table 1; the overall rate was low. Two subjects in the HU group developed acute chest syndrome despite pre-operative transfusion; one episode was likely related to underlying asthma and poor response to hydroxyurea; the second was likely related to pain and hypoventilation after laparoscopic splenectomy and tonsillectomy/adenoidectomy. Overall, there were no significant differences in complications and no significant difference in days to discharge between the two groups. In summary, children with SCA on HU may safely undergo surgery without significantly reducing their percent HbS. Nonetheless, attention should still be made towards multidisciplinary effort to reduce intra- and post-operative complications, and clinicians should consider response to HU, pulmonary status and type of surgery when planning peri-operative management in children with SCA on HU. Disclosures: Off Label Use: hydroxyurea in young children.

scholarly journals Hydroxyurea Prescription Fills and Adherence, Among Pediatric and Adult Medicaid Eligible Patients with Sickle Cell Disease in North Carolina

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3391-3391
Author(s):  
Nirmish Shah ◽  
Christian Douglas ◽  
Nancy Crego ◽  
Emily Bonnabeau ◽  
Marian Earls ◽  
...  
Keyword(s):  
North Carolina ◽  
Sickle Cell Disease ◽  
Old Age ◽  
Sickle Cell ◽  
Research Funding ◽  
Acute Chest Syndrome ◽  
Age Group ◽  
Cell Disease ◽  
Good Adherence ◽  
Number Of Patients

Introduction: Sickle cell disease (SCD) is a complex disease for which pain is the hallmark. Hydroxyurea (HU) is the standard of care for treatment for most patients with SCD and reduces the frequency of pain episodes, acute chest syndrome, need for red blood cell transfusions, hospitalizations and has been shown to improve mortality. Despite National Heart Lung and Blood Institute (NHLBI) recommendations for the use of HU beginning at 9 months of age, adherence has been historically low. We aimed to: 1) describe HU prescription fills and adherence for persons with SCD enrolled in Medicaid during a 12-month period in North Carolina (NC); and 2) determine factors that may predict good adherence. Methods: Medicaid claims were examined from data obtained from Community Care of North Carolina (CCNC) 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) between March 1, 2016 and February 28, 2017. HU claims were identified using the drug name. Only those enrolled in Medicaid for 12 months were included in this analysis. The number of HU prescriptions filled per enrollee by age group was determined by summing the number of filled HU prescriptions over the study period for each eligible enrollee. The number of HU days supplied is the sum of the days of supply on the prescription (e.g. 30-day supply) in a 12-month period per person. The duration of HU treatment days was measured as the number of days between the date of the first HU prescription filled and the last day of the study period. The number of days between breaks in treatment is the sum of days with no HU supplied, divided by the number of gaps (missing next HU prescription fill) per person. HU adherence was categorized into one of the followings: 1) Good - if number of days supplied is ≥80% of duration of HU treatment; 2) Fair or Moderate - if number of days supplied is 60-79% of duration of HU treatment; 3) Poor - if number of days supplied is < 60% of duration of HU treatment. Logistic regression was used to evaluate HU treatment adherence (good versus fair or poor). The model was conditioned on age, gender, participant residence (metro, non-metro adjacent to metro and non-metro non-adjacent to metro), co-management (at least one PCP and one hematologist visit/patient during the study period) and months enrolled in CCNC. Results: A total of 2,790 patients with Medicaid claims data were reviewed, with 649 patients meeting inclusion criteria (at least one HU prescription and 12 months enrollment in Medicaid). The participants in the sample were majority female (51.77%), lived in metropolitan areas (78.12%) and had a mean age of 16.49 years old (SD=11.49) A third of the sample (32.20%) had at least 1 HU prescription during the study period (Table 1). Those who were 1-9 years old had the highest median number of days supplied (221; range 21-750), the least median days between breaks in HU treatment (14.20; range 0-318), and the longest duration of HU treatment days (median 340; range 0-364). Those who were 18-30 years old had the lowest number of median days supplied (110; range 4-366) and the most median days between treatment (49.3; range 0-337). The 1-9 year olds also had the highest number of patients classified as good HU adherence (47.50%) and conversely the lowest classified as poor HU adherence (37.50%). In contrast, the 18-30 year old age group had the lowest good HU adherence (18.03%) and the highest poor HU adherence (69.40%) in the sample. The 31-45 year old age groups had the next lowest good HU adherence (20.93%) and next highest poor HU adherence (60.47%). Good HU adherence was most influenced by participant age. Prediction by co-management was minimal (Figure 1). Gender, residency and number of months enrolled in CCNC had little influence on HU adherence. Conclusions: Although recommended for most patients, HU was prescribed for less than one third of all patients with Medicaid in NC. Pediatric patients had the highest rate of HU prescription (40-46%) and patients over the age of 30 had the lowest (11-12%). In addition, of those prescribed HU, most patients were not classified as having good adherence. Importantly, poor HU adherence was most prevalent in the transition age group (18-30 year old), supporting the need for increased focus during the move from pediatric to adult care. Efforts should continue to explore methods that improve adherence including provider education and innovative patient strategies such as mHealth. Disclosures Shah: Alexion: Speakers Bureau; GBT: Research Funding; Novartis: Consultancy, Research Funding, Speakers Bureau. Tanabe:NIH: Research Funding; AHRQ: Research Funding.

scholarly journals Impact of Sickle Cell Therapies on Growth Patterns

Blood ◽  
2017 ◽  
Vol 130 (Suppl_1) ◽  
pp. 870-870
Author(s):  
Varsha Kulkarni ◽  
Inmaculada Aban ◽  
Krishnaveni Sirigaddi ◽  
Pallavi Iyer ◽  
Jeffrey Lebensburger
Keyword(s):  
Treatment Group ◽  
Sickle Cell ◽  
Research Funding ◽  
Age Effect ◽  
Growth Patterns ◽  
Hemoglobin Level ◽  
Transfusion Therapy ◽  
Significant Difference ◽  
The Impact ◽  
Over Time

Abstract Background: Adolescent patients with Sickle Cell Anemia (SCA) may demonstrate impaired growth. To better understand the impact of transfusion, hydroxyurea, or no modifying therapy on growth, we retrospectively analyzed the growth patterns over 18 years for a large cohort of patients with HbSS or SB0 thalassemia. Methods: We identified 454 subjects with HbSS or SB0 thalassemia in our practice. We recorded growth points (height, weight, body mass index (BMI)) along with white blood cell count, hemoglobin, and SCD modifying therapy from each annual sickle cell visit. For participants that were on transfusion therapy, we recorded the blood count immediately prior to a transfusion. We recorded therapy as the therapy utilized for the majority of visits during the last 12 months. The primary outcome for this project was growth pattern during puberty years. Therefore, we excluded younger participants (&lt;age 8 (girls) or age 9 (boys) or older participants that lacked growth parameters obtained prior to age 8 (girls) or 9 (boys) (total excluded n=233). We analyzed 2119 growth records among the remaining 221 participants. For modeling height over time, we used linear mixed model for other variables with random intercept. This allows us to address the issue of the correlation among repeated measures from the same participant. We fitted the model with age, treatment group and age by treatment group interaction. Treatment effect was deemed significant if the interaction with age was significant. We fitted both linear and quadratic age effect and their interaction with treatment. If there was no evidence of a significant non-linear relationship, we simplified the model to linear age effect. We used a similar approach in modeling the effect of Weight, BMI, and hemoglobin on Height and Treatment. Results: Female participants on chronic transfusion therapy were significantly taller than participants on hydroxyurea or no sickle cell modifying therapy (Figure, p&lt;0.001). There was no significant difference in height over time between females that received hydroxyurea as compared with those that did not receive sickle cell modifying therapies. Male participants on transfusion therapy also were taller than patients who received hydroxyurea or no therapy. (Figure, p&lt;0.001); however, no significant difference in height was observed between participants that were on no SCD modifying therapy as compared to hydroxyurea. Among female participants, we identified a positive relationship between height and hemoglobin level for participants on transfusion but a non-significant increase in height by increasing hemoglobin level for participants on hydroxyurea. Among males, all patients were taller based on hemoglobin level but no significant differences were detected between therapy and hemoglobin level. In analyzing weight for female participants, therapy had a significant impact on weight (p=0.006). Participants on transfusion therapy had increased weight as compared to participants on hydroxyurea or no sickle cell modifying therapy. In males, the overall model again identified therapy as significant for weight gain (p=0.01). Finally, BMI was significantly higher for females participants on transfusion than participants on hydroxyurea or no sickle cell modifying therapy (p=0.01) Among males participants, therapy had a significant impact on BMI (p=0.05) although hydroxyurea participants had the highest BMI. Conclusion: Our data suggests that participants on transfusion therapy are generally taller compared to either hydroxyurea or no SCD modifying therapy. We also suggest that higher hemoglobin is associated with increased growth during pubertal years. Figure Figure. Disclosures Lebensburger: ASH: Research Funding; NHLBI: Research Funding.

scholarly journals Predictors of Maternal Morbidity Among Participants Enrolled in the Sickle Cell Disease Implementation Consortium Registry

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 3-3
Author(s):  
Rita V Masese ◽  
Dominique Bulgin ◽  
Liliana Preiss ◽  
Mitchell Knisely ◽  
Eleanor Stevenson ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Sickle Cell ◽  
Research Funding ◽  
Transfusion Requirement ◽  
The United States ◽  
Acute Chest Syndrome ◽  
Maternal Outcomes ◽  
Maternal Complications ◽  
Advisory Committees ◽  
Increased Risk

Introduction Pregnancy in sickle cell disease (SCD) is associated with an exacerbation of SCD-related complications and an increased risk of maternal complications. The increased risk is partly due to physiologic adaptations in pregnancy, which include increased metabolic demands and a hypercoagulable state. The maternal death rate for SCD is 629 per 100,000 deliveries, compared to 12 per 100,000 deliveries in black women and 6 per 100,000 deliveries in the general population (Raider et al., 2016). Studies on maternal and perinatal outcomes of patients with SCD present inconsistent and conflicting results. Some studies have reported an increase in maternal complications such as pre-eclampsia, acute chest syndrome and thromboembolic events, while other studies have reported no significant risk in adverse maternal outcomes. The inconsistent findings reported in prior studies may be attributed to small sample sizes and single-centered sites. Our study aims to determine the prevalence and predictors of maternal morbidity among participants enrolled in the SCD Implementation Consortium (SCDIC) registry, which is the largest, most geographically diverse SCD participant sample in the United States. Methods This cross-sectional study included women enrolled in the SCDIC registry who had at least one pregnancy event. The SCDIC is composed of eight academic SCD centers across the United States and one data-coordinating center. Participants were enrolled in the SCDIC registry if they were 18 to 45 years of age and had a confirmed diagnosis of SCD. Enrolled participants completed a series of surveys that collected sociodemographic information, SCD and pregnancy history and data abstractions of participants' medical records was completed. Medical complications queried during pregnancy included: vaso-occlusive episodes, acute chest syndrome, blood transfusion requirement, preeclampsia, maternal diabetes and deep venous thrombosis. Descriptive analysis of sociodemographic, clinical and maternal characteristics was conducted. Bivariate analysis was performed using Chi-Square test, Mann-Whitney U test, t-test, and logistic regressions, as appropriate. A p-value of ≤ 0.05 was considered statistically significant for all analysis. Results The study sample included 743 women who had at least one pregnancy event, and a total of 1066 live births. Almost all women (96.3%) were African American, with a median age of 21 years (inter-quartile range of 19 to 23 years) at first birth. The majority had Hb SS SCD genotype (69.5%; 513 of the 738 with SCD genotype data). Of all reported pregnancies, participants did not use hydroxyurea during conception (78%), and pregnancy (84.5%). Only 2.7 % of the women reported using fertility drugs or assisted reproductive procedures. Seventy five percent of the pregnancies that ended in live births had maternal complications. The leading complications were vaso-occlusive episodes (61.2%), pregnancy requiring blood transfusion(s) (33.2%), preeclampsia (15.4%), deep venous thrombosis (5.6%) and acute chest syndrome (7.7%). When the pregnancies were stratified by SCD genotype, women with Hb SS had a higher occurrence of acute chest syndrome (63.4% vs. 26.7%), transfusion requirement (70.8% vs. 21%) and preeclampsia (66.7% vs 22.4%). In the univariate logistic regressions, multiparous women, with a history of adverse maternal outcomes in a previous pregnancy, had higher odds of vaso-occlusive episodes (OR: 3.42; 95% CI: 2.42-4.94) acute chest syndrome (OR:4.99; 95% CI:2.56- 9.48), transfusion requirement (OR:3.86; 95% CI:2.64- 5.69), and pre-eclampsia (OR:3.36; 95% CI:2.05-5.45). Conclusion In this large multicenter registry, we found pregnant women with SCD have significant maternal complications. Early antenatal care by healthcare providers knowledgeable about risk factors for adverse maternal outcomes in SCD is essential improve maternal and fetal outcomes and reduce the maternal death rate for SCD. Disclosures Hankins: Novartis: Research Funding; Global Blood Therapeutics: Consultancy, Research Funding; MJH Life Sciences: Consultancy, Patents & Royalties; UptoDate: Consultancy; National Heart, Lung, and Blood Institute: Honoraria, Research Funding; LINKS Incorporate Foundation: Research Funding; American Society of Pediatric Hematology/Oncology: Honoraria. Treadwell:Global Blood Therapeutics: Consultancy; UpToDate: Honoraria. King:Amphivena Therapeutics: Research Funding; Bioline: Consultancy; Celgene: Consultancy; Cell Works: Consultancy; Incyte: Consultancy; Magenta Therapeutics: Membership on an entity's Board of Directors or advisory committees; Novimmune: Research Funding; RiverVest: Consultancy; Tioma Therapuetics: Consultancy; WUGEN: Current equity holder in private company. Gordeuk:CSL Behring: Consultancy, Research Funding; Global Blood Therapeutics: Consultancy, Research Funding; Imara: Research Funding; Ironwood: Research Funding; Novartis: Consultancy. Kanter:SCDAA Medical and Research Advisory Board: Membership on an entity's Board of Directors or advisory committees; AGIOS: Membership on an entity's Board of Directors or advisory committees; BEAM: Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy; GLG: Honoraria; Jeffries: Honoraria; Cowen: Honoraria; Wells Fargo: Honoraria; NHLBI Sickle Cell Advisory Board: Membership on an entity's Board of Directors or advisory committees; Medscape: Honoraria; Guidepoint Global: Honoraria; bluebird bio, inc: Consultancy, Honoraria; Sanofi: Consultancy. Glassberg:Pfizer: Research Funding; Global Blood Therapeutics: Consultancy; Eli Lilly and Company: Research Funding. Shah:Novartis: Consultancy, Research Funding, Speakers Bureau; Alexion: Speakers Bureau; CSL Behring: Consultancy; Bluebird Bio: Consultancy; Global Blood Therapeutics: Consultancy, Research Funding, Speakers Bureau.

scholarly journals Pregnancies and Neonatal Outcomes in Patients with Sickle Cell Disease (SCD): Still a (High-)Risk Constellation?

2021 ◽  
Vol 11 (9) ◽  
pp. 870
Author(s):  
Pia Proske ◽  
Laura Distelmaier ◽  
Carmen Aramayo-Singelmann ◽  
Nikolaos Koliastas ◽  
Antonella Iannaccone ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Neonatal Outcomes ◽  
University Hospital ◽  
High Rate ◽  
Successful Outcome ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
Significant Difference ◽  
Tract Infections

Background: This monocentric study conducted at the University Hospital of Essen aims to describe maternal and fetal/neonatal outcomes in sickle cell disease (SCD) documented between 1996 to 2021 (N = 53), reflecting the largest monocentric analysis carried out in Germany. Methods/Results: 46 pregnancies in 22 patients were followed. None of the patients died. In total, 35% (11/31) of pregnancies were preterm. 15 pregnancies in eight patients were conceived on hydroxycarbamide (HC), of which nine had a successful outcome and three were terminated prematurely. There was no difference regarding the rate of spontaneous abortions in patients receiving HC compared to HC-naive patients prior to conception. In patients other than HbS/C disease, pregnancies were complicated by vaso-occlusive crises (VOCs)/acute pain crises (APCs) (96%, 23/24); acute chest syndrome (ACS) (13%, 3/24), transfusion demand (79%, 19/24), urinary tract infections (UTIs) (42%, 10/24) and thromboembolic events (8%, 2/24). In HbS/C patients complications included: VOCs/APCs (43%, 3/7; ACS: 14%, 1/7), transfusion demand (14%, 1/7), and UTIs (14%, 1/7). Independent of preterm deliveries, a significant difference with respect to neonatal growth in favor of neonates from HbS/C mothers was observed. Conclusion: Our data support the results of previous studies, highlighting the high rate of maternal and fetal/neonatal complications in pregnant SCD patients.

Sickle crisis in the critically ill

2016 ◽  
Author(s):  
Shilpa Jain ◽  
Mark T. Gladwin
Keyword(s):  
Sickle Cell Disease ◽  
Blood Cell ◽  
Sickle Cell ◽  
Red Blood Cell ◽  
Systemic Infection ◽  
Red Blood Cell Transfusion ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
Ischaemia Reperfusion Injury ◽  
Transfusion Therapy

Sickle cell disease crises are precipitated by an acute occlusion of microvessels, which can lead to end organ ischaemia reperfusion injury and acute haemolysis. Acute fat emboli syndrome, acute lung injury (the acute chest syndrome), acute pulmonary hypertension, and cor pulmonale, haemorrhagic and occlusive stroke, and systemic infection represent the most common life-threatening complications observed in current ICU practice. General principles of management in all patients admitted to the critical care unit are hydration, antibiotics, pain control, and maintenance of oxygenation and ventilation. Red blood cell transfusion therapy is the treatment of choice for most complications of sickle cell disease requiring intensive care management. Transfusion of sickle negative, leukoreduced red blood cells, phenotypically matched for Rhesus and Kell antigens is the minimum standard of care in sickle cell disease patients as they have a high incidence of red blood cell alloimmunization.

An Evidence-Based Approach to the Treatment of Adults with Sickle Cell Disease

Hematology ◽  
2005 ◽  
Vol 2005 (1) ◽  
pp. 58-65 ◽  
Author(s):  
Richard Lottenberg ◽  
Kathryn L. Hassell
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Enzyme Inhibitor ◽  
Controlled Trial ◽  
Acute Chest Syndrome ◽  
Evidence Based ◽  
Clinical Expertise ◽  
Cell Disease ◽  
Transfusion Therapy ◽  
Treatment And Prevention

Abstract The application of evidence-based medicine to the management of adults with sickle cell disease (SCD) is currently primarily driven by clinical expertise and patient preference, as there is a paucity of randomized controlled trial (RCT) data to guide decision-making. A summary of SCD management principles in the areas of health care maintenance, transfusion therapy, treatment and prevention of painful episodes, acute chest syndrome, stroke, renal disease, contraception and pregnancy, and priapism is predominantly based on the authors’ interpretation of available observational studies as well as the opinions of experts in SCD. RCTs impacting current practices address use of hydroxyurea to prevent painful episodes and acute chest syndrome, intensity of pre-operative transfusion, transfusion during pregnancy, and angiotensin-converting enzyme inhibitor therapy for proteinuria, but most issues in adult SCD care have not been rigorously studied and management may not be appropriately extrapolated from pediatric data. While challenging clinical problems need to be addressed by RCTs, there is also the need for development of practice guidelines using formal methodological strategies. This brief review is not a substitute for the process but provides a literature-based approach to making treatment decisions when caring for adults with SCD.

Hospitalization Rates in Patients with Sickle Cell Disease (SCD) in the State of Maryland (MD): No Change Since Approval of Hydroxyurea (HU).

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 107-107
Author(s):  
Sophie Lanzkron ◽  
George J. Dover
Keyword(s):  
Sickle Cell ◽  
Census Data ◽  
Eligible Patient ◽  
Johns Hopkins Hospital ◽  
Acute Chest Syndrome ◽  
Total Hospital ◽  
Significant Difference ◽  
Hospital Days ◽  
Number Of Individuals ◽  
Total Charges

Abstract Background : In 1998 the FDA approved the use of HU for use in patients (pts) with SCD. The trial on which the approval was based demonstrated that adult pts that were on HU had fewer hospitalizations, fewer episodes of acute chest syndrome and required fewer transfusions than those patients that were not on HU. The authors of this original study were able to calculate a cost savings of over 20 million dollars a year if every eligible patient in the US were taking HU. There is currently no literature that addresses the use of HU outside the setting of a clinical study. Methods: We reviewed the inpatient and outpatient charts of all adults with SCD, admitted to Johns Hopkins Hospital (JHH) in FY2003. We also reviewed hospital admission data for MD in FY2003 and FY1995, using data from the MD Health Services Cost Review Commission. Data was pulled using the following ICD9 codes 28260,28261,28263,28269,28262. An estimate of total number of individuals with SCD in MD was made using 1990 and 2000 census data and a prevalence of SCD of 1 in 400 African Americans (AA). Fisher’s exact test was used to compare proportions. As readmission data was unavailable we assumed that the rates of readmission were similar for 1995 and 2003. Results : Review of the JHH data (Table 1) showed that 25% of pts with SCD accounted for half of the hospital days for all pts with SCD and almost 40% of total charges. 66% of eligible pts with Hgb SS were not receiving HU. The reasons for not being on HU varied; 4/9 did not have regular outpatient follow-up, 1 was pregnant, 2 had compliance issues related to side effects, 1 patient refused to take HU and 1 patient started during FY2003. For the pts with hgb SC disease, the indications for use of HU are not established and in our pt cohort those that had received HU in the past did not attain significant benefit without toxicity. Based on US census data the number of individuals of African decent, 18 years and older with SCD in MD for 2003 was 2760. By comparison the number estimated to be in MD in 1995 was 2361. The number of adult admissions for the diagnosis of SCD to hospitals in MD in FY1995 was 1313 and in FY2003 it was 1961. The number of admissions per estimated AA adult with SCD in MD for 1995 was .56 and for 2003 was .71. (p&lt;0.001) The annual costs of caring for hospitalized adult sickle cell pts in MD in 1995 was $6.7 million compared to over $10 million for FY2003. The percent of total hospital expenditures spent on SCD admissions in MD was 0.12% in 1995 and doubled to 0.21% in 2003. Pediatric data over the same time period demonstrated an increase in number of hospitalizations 909 in 1995 compared to 1202 in 2003. When the ratios of admissions per estimated number of pediatric sickle cell pt. in MD were compared for the two time periods there was no statistically significant difference. Conclusions : Hospital generated data for MD demonstrates that admissions and costs of caring for adult sickle cell pts have increased significantly since the approval of HU by the FDA. Data from JHH shows that a large number of eligible patients are not taking HU. Further investigation into the issues affecting hospitalizations of individuals with SCD and the use of HU in this patient population is warranted. Characteristics of SCD Patients Admitted to JHH All SCD patients ≥ 3 admissions/yr (%) Total Patients 109 27(25) Total Hospital days 1266 615(49) Total Admits 225 124 (55) Total Charges ($) 2,796,088 1,090,348(39) Number on HU Unknown 4

Changes of BNP Level and Pulmonary Arterial Pressure during An Acute Sickle Cell Crisis, Comparison with Steady State

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 2503-2503
Author(s):  
Aref Agheli ◽  
Chenthil Rathnasabapathy ◽  
Ashish Sangal ◽  
Zili He ◽  
William Steier ◽  
...  
Keyword(s):  
Steady State ◽  
Right Ventricular ◽  
Sickle Cell ◽  
Ventricular Dysfunction ◽  
Right Ventricular Dysfunction ◽  
Acute Chest Syndrome ◽  
Cardiac Complications ◽  
Significant Difference ◽  
Sickle Cell Crisis ◽  
Pulmonary Arterial

Abstract Background: The heart is frequently involved in Sickle Cell Anemia (SCA). Cardiomegaly is a usual finding, significant arrythmias and sudden death are common, and 30% of patients with both homozygous and heterozygous SCA develop Pulmonary Arterial Hypertension (PAH), a major risk factor for higher mortality in this population. Brain Natriuretic Peptide (BNP) and echocardiographic data could provide important prognostic and diagnostic information about PAH in SCD. High levels of BNP, which is released from ventricular cardiomyocytes in response to their stretch, reflect cardiac chamber volume and pressure overload in various conditions. In patients with PAH, BNP levels correlate with the severity of Pulmonary Artery Pressure (PAP) elevation and right ventricular dysfunction. In human, the half life of BNP is 20 minutes, reflecting the fluctuation of BNP levels during different stages of any acute cardiac pathology. Methodology: The hypothesis of this prospective IRB approved study was to investigate the BNP level and PAP elevation during an acute Sickle Cell Crisis (SCC), in particular in those with intrathoracic structures involvement. Between December 2006 and July 2008, 81 patients were registered after a written informed consent was obtained. We collected the BNP levels and echocardiographic data of patients with SCD and compared them in two group; those who were admitted with Sickle Cell Crisis (SCC) and those who returned to clinic in Steady State (SS) for follow up. The data were obtained on the first day of admission in SCC group. The primary endpoint was the elevation of the BNP level and the secondary endpoint was elevation of the PAP during a SCC, which were compared with SS patients. The inclusion criterion was age above 18 and having one of the sickle cell syndromes, requiring hospital admission. Results: Forty nine patients (59%) were female, and 34 (41%) patients were male. Their ages ranged from 19 to 65, mean (SD) 30.2 (9.7) years. The mean (SD) levels of BNP were significantly higher in patients who were admitted with one of the acute complications or vaso-occlusive crisis of sickle cell, [177.3 (23.4) pg/ml], when compared with its levels in SS, [34.17 (6.1) pg/ml], (95% CI 61.4 to 225.0, p&lt;.001) (Figure 1). An elevated BNP level was defined as levels more than 100 pg/ml. A further subgroup analysis revealed that the BNP levels were even more significantly higher in patients with acute chest syndrome or other intrathoracic events [(n= 17, mean (SD) 363.6 (121.3) pg/ml], when compared with those of simple acute sickle cell crisis, [(n= 35, mean (SD) 167.7 (26.8) pg/ml] (p=.038) (Figure 1). Topographic data about heart chambers’ sizes, volumes, and pressures were obtained by Echocardiography and compared in two groups. While only 23.1% of patients in SS group had elevated PAP with a mean (SD) of 43 (2.1) mmHg, 41.1% (n=21) of patients with SCC had elevated PAP with mean (SD) 45.9 (2.1) mmHg, with no significant difference between two groups with PAH (p=.608). Conclusion: Patients with either homozygous or heterozygous forms of SCA can have cardiac complications, such systolic and diastolic dysfunction. Hypoxemia leads to raised levels of BNP production. In patients with SCA, an elevated BNP level largely reflects the severity of right ventricular dysfunction associated with PAH. Our data revealed that BNP level and PAP are increased during vaso-occlusive crisis of SCA, in particular during those life-threatening complications, such acute chest syndrome. These changes seem to be temporary and with clinical improvement, the majority of patients’ BNP levels and PAP return to the baseline, although some will never normalize. Figure Figure

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