scholarly journals Comparison of Clinical Evolution of Children with Sickle Cell Disease before and after Treatment with Hydroxyurea at Saint Damien Hospital, Tabarre-Haiti,2013-2018

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2306-2306
Author(s):  
Nora St Victor Dély ◽  
Ofelia A. Alvarez ◽  
Vanessa J Dor ◽  
Emmeline Lerebours
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Clinical Course ◽  
Acute Chest Syndrome ◽  
Blood Transfusions ◽  
Cell Disease ◽  
Clinical Evolution ◽  
Before And After ◽  
The Mean ◽  
Painful Crisis

INTRODUCTION Sickle cell disease (SCD), an autosomal recessive hemoglobinopathy, is associated with a high morbidity and mortality rate, especially in low income countries. In Africa, 5% of deaths among children under five are attributable to SCD [59th World Health Assembly, WHO 2006]. This chronic disease greatly alters the quality of life of affected children. However, according to several published studies, SCD clinical course can be improved with the administration of hydroxyurea, an antimetabolite drug. [Nkashama, Pan African Medical Journal,2015] Saint-Damien, a pediatric hospital in Haiti, has a current cohort of 1248 sickle cell children. Forty of them (3 %) benefit from hydroxyurea administration since November 2015. In this hospital, data on how hydroxyurea modifies SCD clinical course are lacking, despite the advantage of this drug described in literature [Charache,New England Journal of Medicine,1995]. This study aims to compare the evolution of children treated at Saint Damien Hospital, before and after receiving hydroxyurea. METHODS A retrospective analytic study was conducted from November 2013 to June 2018 in the Sickle Cell Clinic at Saint-Damien Hospital. We included 40 children aged 2 to 15 years old treated with hydroxyurea. All of them benefit of the same treatment protocol: Initial dose of 10 mg per kg per day increase to maintenance dose of 25 mg per kg per day. Any child whose treatment has been permanently discontinued regardless of the cause was excluded. Epidemiological and clinical data were collected using Excel 2010. We compared children clinical evolution two years before and two years after hydroxyurea administration using these parameters: frequency and duration of hospitalizations, hospitalization frequency for specific complications (pain crisis, stroke and acute chest syndrome), and frequency of blood transfusions. We calculated frequencies, ratios and means using Epi Info. We realized statistical analysis to compare quantitative variables with a p value significant when less than 5%. RESULTS Gender ratio was 1:1. The mean age of children at enrollment on hydroxyurea was 8 years. Thirty-eight children of 40 (95 %) experienced at least one hospitalization before receiving the drug, compared with 17 (42.5%) after, p=0.025. The mean duration of hospitalization was 9 days before and 6 days after, p=0.0319. The average number of hospitalizations per child was decreased by 30 %. Seventy percent of children were hospitalized at least once due to painful crisis 2 years before receiving hydroxyurea, compare to 22.5 % after. Thirty-one children (77.5%) were transfused at least once before receiving the drug and 9 (22.5%) after receiving it. There was no cases of acute chest syndrome or stroke reported after hydroxyurea, unlike before the introduction of the drug. (Table 1) CONCLUSION The percentage of hospitalized children and the average length of hospitalization stay decreased significantly with hydroxyurea intake; as well as the frequency of painful crisis and blood transfusions. Hydroxyurea acts directly on the two main causes of hospitalization in the sickle cell, reducing the morbidity related to this pathology; and demonstrating the direct benefit of this drug at Saint Damien Hospital. Since our cohort is young, we have not been able to follow his evolution over a longer period of time. We plan to continue to observe this cohort. But these first results already allow us to recommend a broader use of hydroxyurea for pediatric patients with SCD in Haiti. Disclosures Alvarez: Forma Therapeutics: Consultancy; Novartis: Consultancy.

scholarly journals Hospitalization Events among Children and Adolescents with Sickle Cell Disease in Basra, Iraq

Anemia ◽  
2015 ◽  
Vol 2015 ◽  
pp. 1-8 ◽  
Author(s):  
Zeina A. Salman ◽  
Meaad K. Hassan
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Readmission Rate ◽  
Length Of Hospital Stay ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
Opioid Use ◽  
Asthma Symptoms ◽  
The Mean ◽  
High Readmission Rate

Objectives. Despite improvements in the management of sickle cell disease (SCD), many patients still experience disease-related complications requiring hospitalizations. The objectives of this study were to identify causes of hospitalization among these patients and factors associated with the length of hospital stay (LOS) and readmission.Methods. Data from 160 patients (<14 years old) with SCD who were admitted to the Basra Maternity and Children’s Hospital from the first of January 2012 through July 2012 were analyzed.Results. The main causes of hospitalization were acute painful crises (73.84%), infections (9.28%), acute chest syndrome (8.02%), and acute splenic sequestration crisis (6.32%). The mean LOS was4.34±2.85days. The LOS for patients on hydroxyurea (3.41±2.64days) was shorter than that for patients who were not (4.59±2.86days),P<0.05. The readmission rate (23.1%) was significantly higher among patients with frequent hospitalizations in the previous year (OR 9.352, 95% CI 2.011–43.49), asthma symptoms (OR 4.225, 95% CI 1.125–15.862), and opioid use (OR 6.588, 95% CI 1.104–30.336). Patients on hydroxyurea were less likely to be readmitted (OR 0.082, 95% CI 0.10–0.663).Conclusions. There is a relatively high readmission rate among patients with SCD in Basra. The use of hydroxyurea significantly decreases the LOS and readmission rate.

scholarly journals Beneficial effects of adenotonsillectomy in children with sickle cell disease

2020 ◽  
Vol 6 (4) ◽  
pp. 00071-2020
Author(s):  
Ilaria Liguoro ◽  
Michele Arigliani ◽  
Bethany Singh ◽  
Lisa Van Geyzel ◽  
Subarna Chakravorty ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Arterial Oxygen Saturation ◽  
Arterial Oxygen ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
Beneficial Effects ◽  
Admission Rates ◽  
The Mean ◽  
The Impact

Tonsillectomy and adenoidectomy (T&A) is frequently performed in children with sickle cell disease (SCD). Our aim was to evaluate the impact of this surgery on overnight oxygenation and rates of complications in these patients.Children with SCD who underwent T&A between 2008 and 2014 in two tertiary hospitals were retrospectively evaluated. Overnight oximetry and admission rates due to vaso-occlusive pain episodes (VOEs) and acute chest syndrome (ACS) in the year preceding and following the surgery were compared.19 patients (10 males, 53%) with a median age of 6 years (range 3.5–8) were included. A significant increase of mean overnight arterial oxygen saturation measured by pulse oximetry (SpO2) (from 93±3.6% to 95.3±2.8%, p=0.001), nadir SpO2 (from 83.0±7.1% to 88±4.1%, p=0.004) and a reduction of 3% oxygen desaturation index (from a median value of 5.7 to 1.8, p=0.003) were shown. The mean annual rate of ACS decreased from 0.6±1.22 to 0.1±0.2 events per patient-year (p=0.003), while the mean cumulative rate of hospitalisations for all causes and the incidence of VOEs were not affected.T&A improved nocturnal oxygenation and was also associated with a reduction in the incidence of ACS at 1-year follow-up after surgery.

scholarly journals Clinical events in the first decade in a cohort of infants with sickle cell disease. Cooperative Study of Sickle Cell Disease [see comments]

Blood ◽  
1995 ◽  
Vol 86 (2) ◽  
pp. 776-783 ◽  
Author(s):  
FM Gill ◽  
LA Sleeper ◽  
SJ Weiner ◽  
AK Brown ◽  
R Bellevue ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Clinical Course ◽  
The United States ◽  
Beta Thalassemia ◽  
Acute Chest Syndrome ◽  
Cooperative Study ◽  
Cell Disease ◽  
Splenic Sequestration ◽  
Clinical Events

Within the Cooperative Study of Sickle Cell Disease, 694 infants with confirmed sickle cell disease were enrolled at less than 6 months of age. Information about the nature and frequency of complications was collected prospectively over a 10-year period. Painful crises and acute chest syndrome were the most common sickle cell-related events in homozygous sickle cell anemia (SS), hemoglobin SC disease (SC), and S beta thalassemia patients (overall incidence in SS patients of 32.4 and 24.5 cases per 100 person-years, respectively). Bacteremia occurred most frequently in SS children under 4 years of age and in SC patients less than 2 years of age. The mortality rate was low in this cohort compared with that found in previous reports. Twenty children, all with Hb SS, died (1.1 deaths per 100 person-years among SS patients). Infection, most commonly with Streptococcus pneumoniae and Hemophilus influenzae, caused 11 deaths. Two children died of splenic sequestration, 1 of cerebrovascular accident, and 6 of unclear causes. Two patients underwent cholecystectomies, and 17 underwent splenectomies after one or more splenic sequestration crises. The experience of this cohort should reflect closely the true clinical course of those children with Hb SS and Hb SC disease who are observed in sickle cell centers in the United States.

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.

scholarly journals Gene-centric association study of acute chest syndrome and painful crisis in sickle cell disease patients

Blood ◽  
2013 ◽  
Vol 122 (3) ◽  
pp. 434-442 ◽  
Author(s):  
Geneviève Galarneau ◽  
Sean Coady ◽  
Melanie E. Garrett ◽  
Neal Jeffries ◽  
Mona Puggal ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Association Study ◽  
Sickle Cell ◽  
Genome Wide Association Study ◽  
Inflammatory Responses ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
Genome Wide ◽  
Key Points ◽  
Painful Crisis

Key Points Using genome-wide association study, we found the first replicated genetic association with acute chest syndrome in sickle cell disease patients. The locus identified includes COMMD7, a gene highly expressed in the lung that interacts with NFκB to control inflammatory responses.

Outcomes of Chronic Automated RBC Exchange Program in Omaha, Nebraska 2015-2020

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 21-22
Author(s):  
Aleh Bobr ◽  
Scott A Koepsell ◽  
Julie Eclov ◽  
Omar Abughanimeh ◽  
Steven Ebers ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Blood Cell ◽  
Iron Overload ◽  
Sickle Cell ◽  
Red Blood Cell ◽  
Beta Thalassemia ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
Before And After ◽  
Ed Visits

Background: Red blood cell exchange (RBCX) is an effective therapy in the treatment of different hemoglobinopathies. The University of Nebraska Medical Center (UNMC) established a chronic RBCX program in November 2015, which took care of patients with multiple hemoglobinopathies. In this study, we aim to evaluate the outcomes of this program. Methods: This is a retrospective study. After an IRB approval, we reviewed the charts of patients who were enrolled in the chronic RBCX program between 11/2015-7/2020 at UNMC. Data was collected to evaluate indications of RBCX, types of hemoglobinopathies, hemoglobinopathies' complications before and after the enrollment in the program, and assessment of hospital visits before and after enrollment in the program. Results: In November 2015, the chronic RBCX program was established in Nebraska. Since the start, 24 patients came through the program and 20 patients are still actively enrolled and undergoing regular exchange transfusions. The four patients who left the program did it for the following reasons: moving out of state, stem cell transplant and change to different treatment modality. Four of 24 patients were beta thalassemia patients (two of them with combined HbE/beta thalassemia). Twenty patients had sickle cell disease with two of them having combined beta thalassemia and HbS and one with alpha thalassemia and HbS. The indications ranged from history of stroke, intracranial vascular stenosis, acute chest syndrome (ACS), iron overload, multiple vascular occlusive crises (VOC) and intolerance of medications with most of the patients having multiple indications from the list above (Figure 1). There are several positive outcomes from being on the program. In the patients who had been on the program for at least one year (n=11), nine started the program with iron overload and all of them had a significant decrease in serum ferritin (average 751 ng/mL) with three patients returning to normal range. In the patients who had been in the program at least six months (n=16), 13 patients started with iron overload with five returning to normal range and average decrease in ferritin of 585 ng/mL. Another positive outcome is the number of emergency department (ED) visits for pain crisis. We noted reduction in ED visits in all patients who were in the program for at least six months (n=14), with the exception of one patient where the visits were likely the part of drug seeking behavior. In fact 12 of 13 patients had one or no ED visits within one year after starting on the chronic exchange program having had from 2-11 visits a year prior. None of the patients in the program experienced more severe complications of sickle cell disease, like stroke and acute chest syndrome, while on the program. Due to high volumes of transfusion, there is a big concern about developing red blood cell antibodies in sickle cell disease patients who in general have higher red blood cell antibody burden. Out of 24 patients in the program, six had pre-existing antibodies. For the duration of the program, no new alloantibodies were discovered in the chronically exchanged patients despite high transfusion volumes (range 14L-30L/year). The transfused blood was matched for Rh and Kell antigens for the patients with no antibody history. The patients with previous antibody history had additional matching for the antigen to which antibody was directed. Conclusion:Automated chronic RBCX transfusion program is safe to perform. It leads to significant reduction in volume overload and ED visits. Performing high volume transfusions outside of acute sickle cell crisis and with Rh and Kell matched units prevents formation of RBC antibodies Disclosures Gundabolu: BioMarin:Consultancy;Bristol Myers Squibb pharmaceuticals:Consultancy.

Progression of Pulmonary Hypertension in Patients with Sickle Cell Disease.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 3187-3187 ◽  
Author(s):  
Kenneth I. Ataga ◽  
Charity Moore ◽  
Susan Jones ◽  
Oludamilola Olajide ◽  
Dell Strayhorn ◽  
...  
Keyword(s):  
Pulmonary Hypertension ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Left Ventricular ◽  
Right Atrial ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
Original Cohort ◽  
The Mean

Abstract Introduction: The prevalence of pulmonary hypertension (PHT) is high in patients with sickle cell disease (SCD). Although most patients have only mild increases in their pulmonary artery systolic pressure (PASP), the presence of PHT is strongly associated with an increased risk of death. While PHT seen in SCD is thought to progress over time, both the rate of development of PHT and the factors that affect disease progression remain unknown. Methods: The 41 subjects in this study were drawn from an original cohort of 60 patients followed in the Sickle Cell Clinic at UNC-Chapel Hill. All patients were previously evaluated for PHT (defined using an age-, sex-, and BMI-adjusted reference range). Of the 60 patients in the original cohort, six are now deceased and 13 others were not available for repeat evaluation. The PASP was determined using Doppler echocardiography and then applying the modified Bernoulli equation (PASP = 4V2 + right atrial pressure). Individuals were not studied if they: 1) showed clinical evidence of left ventricular failure; 2) had a recent acute illness (e.g., vaso-occlusive crisis); or 3) had experienced an episode of acute chest syndrome within the preceding 4 weeks. Means and standard deviations were calculated for all measures at the time of initial evaluation and at the time of follow-up. Results: Of the 41 subjects in our study, PHT was originally present in 12, while no evidence of PHT was present in 29. Of the 29 subjects who initially had no evidence of PHT, 4 (or 14%) have now developed PHT (mean follow-up period of 3.3 ± 0.4 years). In these 4 subjects, the mean PASP at the time of initial and follow-up evaluations respectively were: 37.0 ± 2.0 mm Hg vs. 55.8 ± 11.0 mm Hg. The patients who developed PHT during the course of the study had lower systolic BP (143 ± 12 mm Hg vs. 128 ± 12 mm Hg), lower fetal hemoglobin levels (6.2 ± 5.7 % vs. 4.2 ± 3.7 %), and higher platelet counts (276 ± 119 X 103/μL vs. 426 ± 96 X 103/μL) at the time of their follow-up analyses. By contrast, 3 of the 12 subjects (or 25%) who were thought to have PHT at the time of their original evaluations were found to have normal PASP determinations at the time of their repeat echocardiograms (mean follow-up period of 3.2 ± 0.6 years). In these latter 3 subjects, the mean PASP values at the time of the initial and follow-up evaluations respectively were: 40.0 ± 4.6 mm Hg vs. 33.7 ± 4.7 mm Hg. Conclusion: In this small group of patients with SCD, we found that PHT developed in 14% of subjects who had no evidence of PHT 3 years earlier. Based on this observation, it seems that periodic echocardiograms to screen for the development of PHT would be appropriate. On the other hand, our observation that some patients initially classified as having PHT failed to have elevated PASP measurements at the time of follow-up illustrates the limitation of a single echocardiographic evaluation in establishing this diagnosis. Because of the increase in PASP that occurs during acute vaso-occlusive episodes, and the difficulty usually encountered in distinguishing steady state from crisis, the initial elevation of the PASP in these patients could have resulted from sub-clinical crisis states. For these reasons, a patient found to have an elevated PASP at the time of a screening echocardiogram should have a repeat study, and perhaps a right heart catheterization, before the diagnosis of PHT is firmly established.

The Use of Chronic Transfusions in Sickle Cell Disease for Non-Stroke Related Indications

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 4934-4934 ◽  
Author(s):  
Lindsay Mize ◽  
Shelly Burgett ◽  
Julia Xu ◽  
Jennifer Rothman ◽  
Nirmish Shah
Keyword(s):  
Sickle Cell Disease ◽  
Acute Care ◽  
Sickle Cell ◽  
Emergency Department Visits ◽  
Acute Chest Syndrome ◽  
Blood Transfusions ◽  
Recurrent Pain ◽  
Cell Disease ◽  
Pain Crisis

Abstract Introduction: Sickle cell disease (SCD) is a chronic disease that can cause significant complications including acute chest syndrome, recurrent pain and stroke. Current guidelines for the use of chronic transfusions include primary and secondary prevention of stroke. Although there is currently limited support for the routine use of transfusions for acute vaso-occlusive crisis (VOC), there has been increasing use of chronic transfusions as an alternative treatment for recurrent VOC. Moreover, there is evidence that patients on chronic transfusions have less VOC. We sought to review the outcomes of patients at our institution placed on chronic transfusions for non-stroke related indications. Methods: We performed a retrospective cohort study to summarize clinical and nonclinical features of sickle cell patients on transfusions for non-stroke related complications. Demographic, clinical, and laboratory information were summarized. Acute care events per month were calculated for both the year prior and up to one year following initiation of chronic transfusions. Acute care events were defined as emergency department visits or hospitalization. Results: Of the 378 patients with SCD treated in the pediatric specialty clinic, there were 21 patients being either chronically transfused or exchange transfused. Six (20%) of these patients were initiated on chronic blood transfusions (CBT) for recurrent pain crisis (median age = 12, range 8 to 17). One of these patients also had suspected hepatic sequestration. All patients were type SS and had been treated with hydroxyurea (HU) for an average length of 6.5 years (range 1 to 12 years) at a mean dose of 25 mg/kg (SD 4) prior to initiation of CBT. All patients continued on HU during chronic blood transfusions. Patients were on chronic transfusions for a median of 11 months (range 3 to 58 months) with mean %S while on transfusions of 39.6% (SD 10). Patients were transfused on average every 5 weeks (range 4 to 6 weeks). Following initiation of transfusions, 50% were started on chelation based on criteria of having a ferritin >1000 ng/mL. Mean peak ferritin was not significantly increased in the year following the start of CBT (513 ng/mL ± 343 vs. 1260 ± 934, p=0.13). There was one new alloantibodies (anti-Jk) reported following initiation of CBT, which developed within 3 months. Acute care visits per month were significantly higher in the year prior as compared to after initiation of chronic blood transfusions (1.04 ± 0.45 vs. 0.28 ± 0.22, respectively; p=0.006) (Figure 1). Discussion: We found that patients started on chronic transfusions for pain crisis had a non-significant increase in peak ferritin and a significant reduction in acute care visits. Prior to CBT, all patients had been initiated on hydroxurea (mean dose of 25mg/kg) in an attempt to treat recurrent VOC. However, following therapy for an average of 6.5 years, patients were placed on CBT to prevent further acute care visits and reduce morbidity. All patients were continued on HU while on CBT with no dose adjustment or effort to titrate to maximum tolerated dose. While on CBT, patients had a mean %S of 40%, which is higher than the recommended goal of 30% for stroke related indications. Importantly, despite the higher mean %S, there was a drastic and significant decrease in acute care visits. It should be noted that although only three patients (50%) of patients were placed on chelation, the remaining three had been on transfusions for less than or equal to 6 months and likely to require chelation with continued therapy. The expected elevated ferritin highlights the difficulty in long-term treatment with chronic transfusion and risk for eventual iron overload. The balance between the clinical benefit and potential long-term complications leads to individual assessment of the risks and benefits prior to initiation of chronic transfusions for VOC. These results advocate for the use of prospective studies to evaluate the role for chronic transfusions for non-stroke related indications in SCD. Figure 1 Figure 1. Disclosures Shah: Novartis: Speakers Bureau.

scholarly journals Treatment patterns and economic burden of sickle-cell disease patients prescribed hydroxyurea: a retrospective claims-based study

2019 ◽  
Vol 17 (1) ◽  
Author(s):  
Nirmish Shah ◽  
Menaka Bhor ◽  
Lin Xie ◽  
Rashid Halloway ◽  
Steve Arcona ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Economic Burden ◽  
Medication Possession Ratio ◽  
Treatment Patterns ◽  
Care Utilization ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
The Mean

Abstract Background This study aimed to evaluate sickle-cell disease (SCD) treatment patterns and economic burden among patients prescribed hydroxyurea (HU) in the US, through claims data. Methods SCD patients with pharmacy claims for HU were selected from the Medicaid Analytic Extracts (MAX) from January 1, 2009 - December 31, 2013. The first HU prescription during the identification period was defined as the index date and patients were required to have had continuous medical and pharmacy benefits for ≥6 months baseline and 12 months follow-up periods. Patient demographics, clinical characteristics, treatment patterns, health care utilization, and costs were examined, and variables were analyzed descriptively. Results A total of 3999 SCD patients prescribed HU were included; the mean age was 19.24 years, most patients were African American (73.3%), and the mean Charlson comorbidity index (CCI) score was 0.6. Asthma (20.3%), acute chest syndrome (15.6%), and infectious and parasitic diseases (20%) were the most prevalent comorbidities. During the 12-month follow-up period, 58.9% (N = 2357) of patients discontinued HU medication. The mean medication possession ratio (MPR) was 0.52, and 22.3% of patients had MPR ≥80%. The average length of stay (LOS) for SCD-related hospitalization was 13.35 days; 64% of patients had ≥1 SCD-related hospitalization. The mean annual total SCD-related costs per patient were $27,779, mostly inpatient costs ($20,128). Conclusions Overall, the study showed the patients had significant unmet needs manifest as poor medication adherence, high treatment discontinuation rates, and high economic burden.

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