Financial analysis of chronic transfusion for stroke prevention in sickle cell disease

Blood ◽  
2000 ◽  
Vol 96 (7) ◽  
pp. 2369-2372 ◽  
Author(s):  
Alan S. Wayne ◽  
Steve E. Schoenike ◽  
Charles H. Pegelow
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Stroke Prevention ◽  
Financial Analysis ◽  
Allogeneic Bone Marrow Transplantation ◽  
Financial Impact ◽  
Red Blood Cell Transfusion ◽  
Allogeneic Bone ◽  
Cell Disease ◽  
Transfusion Therapy

Abstract Chronic red blood cell transfusion can prevent many of the manifestations of sickle cell disease. The medical costs of chronic transfusion and management of associated side effects, especially iron overload, are considerable. This study was undertaken to evaluate the financial impact of chronic transfusion for stroke prevention in patients with sickle cell anemia. Outpatient charges pertaining to hospital-based Medicare uniform bill (UB-92) codes, professional fees, and iron chelation were evaluated. Data were collected on 21 patients for a total of 296 patient months (mean, 14; median, 14 months/patient). Charges ranged from $9828 to $50 852 per patient per year. UB-92, chelation, and physician-related charges accounted for 53%, 42%, and 5% of total charges, respectively. Of UB-92 charges, 58% were associated with laboratory fees and 16% were related to the processing and administration of blood. Charges for patients who required chelation therapy ranged from $31 143 to $50 852 per patient per year (mean, $39 779; median, $38 607). Deferoxamine accounted for 71% of chelation-related charges, which ranged from $12 719 to $24 845 per patient per year (mean, $20 514; median, $21 381). The financial impact of chronic transfusion therapy for sickle cell disease is substantial with charges approaching $400 000 per patient decade for patients who require deferoxamine chelation. These data should be considered in reference to cost and efficacy analyses of alternative therapies for sickle cell disease, such as allogeneic bone marrow transplantation.

Financial analysis of chronic transfusion for stroke prevention in sickle cell disease

Blood ◽  
2000 ◽  
Vol 96 (7) ◽  
pp. 2369-2372
Author(s):  
Alan S. Wayne ◽  
Steve E. Schoenike ◽  
Charles H. Pegelow
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Stroke Prevention ◽  
Financial Analysis ◽  
Allogeneic Bone Marrow Transplantation ◽  
Financial Impact ◽  
Red Blood Cell Transfusion ◽  
Allogeneic Bone ◽  
Cell Disease ◽  
Transfusion Therapy

Chronic red blood cell transfusion can prevent many of the manifestations of sickle cell disease. The medical costs of chronic transfusion and management of associated side effects, especially iron overload, are considerable. This study was undertaken to evaluate the financial impact of chronic transfusion for stroke prevention in patients with sickle cell anemia. Outpatient charges pertaining to hospital-based Medicare uniform bill (UB-92) codes, professional fees, and iron chelation were evaluated. Data were collected on 21 patients for a total of 296 patient months (mean, 14; median, 14 months/patient). Charges ranged from $9828 to $50 852 per patient per year. UB-92, chelation, and physician-related charges accounted for 53%, 42%, and 5% of total charges, respectively. Of UB-92 charges, 58% were associated with laboratory fees and 16% were related to the processing and administration of blood. Charges for patients who required chelation therapy ranged from $31 143 to $50 852 per patient per year (mean, $39 779; median, $38 607). Deferoxamine accounted for 71% of chelation-related charges, which ranged from $12 719 to $24 845 per patient per year (mean, $20 514; median, $21 381). The financial impact of chronic transfusion therapy for sickle cell disease is substantial with charges approaching $400 000 per patient decade for patients who require deferoxamine chelation. These data should be considered in reference to cost and efficacy analyses of alternative therapies for sickle cell disease, such as allogeneic bone marrow transplantation.

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.

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

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

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

scholarly journals Silent cerebral infarcts occur despite regular blood transfusion therapy after first strokes in children with sickle cell disease

Blood ◽  
2011 ◽  
Vol 117 (3) ◽  
pp. 772-779 ◽  
Author(s):  
Monica L. Hulbert ◽  
Robert C. McKinstry ◽  
JoAnne L. Lacey ◽  
Christopher J. Moran ◽  
Julie A. Panepinto ◽  
...  
Keyword(s):  
Magnetic Resonance ◽  
Sickle Cell Disease ◽  
Blood Transfusion ◽  
Sickle Cell ◽  
Stroke Prevention ◽  
Secondary Stroke Prevention ◽  
Cell Disease ◽  
Cerebral Infarcts ◽  
Eligibility Criteria ◽  
Transfusion Therapy

Abstract Children with sickle cell disease (SCD) and strokes receive blood transfusion therapy for secondary stroke prevention; despite this, approximately 20% experience second overt strokes. Given this rate of second overt strokes and the clinical significance of silent cerebral infarcts, we tested the hypothesis that silent cerebral infarcts occur among children with SCD being transfused for secondary stroke prevention. A prospective cohort enrolled children with SCD and overt strokes at 7 academic centers. Magnetic resonance imaging and magnetic resonance angiography of the brain were scheduled approximately every 1 to 2 years; studies were reviewed by a panel of neuroradiologists. Eligibility criteria included regularly scheduled blood transfusion therapy. Forty children were included; mean pretransfusion hemoglobin S concentration was 29%. Progressive cerebral infarcts occurred in 45% (18 of 40 children) while receiving chronic blood transfusion therapy; 7 had second overt strokes and 11 had new silent cerebral infarcts. Worsening cerebral vasculopathy was associated with new cerebral infarction (overt or silent; relative risk = 12.7; 95% confidence interval, 2.65-60.5, P = .001). Children with SCD and overt strokes receiving regular blood transfusion therapy experience silent cerebral infarcts at a higher rate than previously recognized. Additional therapies are needed for secondary stroke prevention in children with SCD.

scholarly journals Effects of Combination Hydroxyurea and Chronic Red Blood Cell Transfusion Therapy on the Immune Parameters of Patients with Sickle Cell Disease

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4840-4840
Author(s):  
Mahogany Oldham ◽  
Gelina Sani ◽  
Stefanie Margulies ◽  
Jennifer Webb ◽  
Robert Sheppard Nickel ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Combination Therapy ◽  
Blood Cell ◽  
Nk Cells ◽  
Sickle Cell ◽  
Red Blood Cell ◽  
Nk Cell ◽  
Red Blood Cell Transfusion ◽  
Cell Disease ◽  
Transfusion Therapy

Background: Sickle cell disease (SCD) is typically characterized as a red blood cell disorder but our understanding of the effects on the immune system is limited. Patients with sickle cell disease have been shown to have unique inflammatory profiles, immune phenotypes and function. Others have shown that during vaso-occlusive crises patients with SCD have elevated counts of neutrophils, monocytes, and cytokines as well as increased activity of invariant natural killer T cells (iNKT).We have previously shown that hydroxyurea use is associated with a normalization of the increased NK cell number and function. While there are studies that describe on the effects of single therapy, there is little known about combination therapy. Therefore, our study investigated immunological changes in pediatric patients on combination therapy, which was defined as hydroxyurea added to chronic red blood cell transfusion treatment. Methods: Patient data and peripheral blood samples were collected from an ongoing pilot study of combination therapy hydroxyurea and simple chronic transfusion in patients with SCD previously on chronic transfusion for stroke prevention. A total of 11 patients with hemoglobin SS were studied at two time points; baseline (on chronic RBC transfusion only) and 3 months follow up after initiation of hydroxyurea 20 mg/kg/day. Comparisons were performed using paired t-tests with a p-value <0.05 being considered significant. Results: T, B and NK cell percentage was similar between baseline and after 3 months of combination therapy 62.5% (44.3-71.9) vs 67% (17.6-82.7), 16.29%(8.15-30.2) vs. 13.26% (1.07-32.8) and 7.79% (4.16-14.7) vs. 6.88 (1.54-21) (p>0.05). There were no significant differences between markers of NK cell activation between baseline and 3 months as follows: NKG2D 4.89% (0.47-28.4) vs. 24.38% (0.88-63), and NKp30 8.40% (0.81-58.7) vs. 32.42% (0.45-86.9). However there was a significant decrease in the percentage of mature (CD57+) NK cells 33.8% (10.7-67.6) vs. 23.07%(4.23-37), p =0.005. Similar results were also seen when using absolute values of the different lymphocyte subsets. Conclusion: Combination therapy appears to not affect overall percentages of B, T and NK cells but does appear to decrease the percentage of mature CD57+ NK cells that are known to have increased cytolytic activity. We plan to investigate the implications of these findings using NK functional studies such as cytotoxicity assays and cytotoxic granule release to further elucidate if combination therapy can lead to a decrease in NK cell function to normal levels. Additionally we plan to assess the effect on the immune parameters at 1 year as the hydroxyurea effect is likely time-dependent. These findings may have implications for patients on chronic transfusion therapy who plan to undergo bone marrow transplantation where a reduction in the potential for graft rejection by NK cells is desired. Disclosures No relevant conflicts of interest to declare.

Live birth after ovarian tissue autograft in a patient with sickle cell disease treated by allogeneic bone marrow transplantation

2010 ◽  
Vol 93 (7) ◽  
pp. 2413.e15-2413.e19 ◽  
Author(s):  
Christophe Roux ◽  
Clotilde Amiot ◽  
Germain Agnani ◽  
Yves Aubard ◽  
Pierre-Simon Rohrlich ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Live Birth ◽  
Marrow Transplantation ◽  
Ovarian Tissue ◽  
Allogeneic Bone Marrow Transplantation ◽  
Allogeneic Bone ◽  
Cell Disease

Encephaloduroarteriosynangiosis (EDAS) For Patients With Sickle Cell Disease

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 999-999
Author(s):  
Jeffrey D. Lebensburger ◽  
Christina J. Bemrich-Stolz ◽  
Christoph Griessenauer ◽  
Lee Hilliard ◽  
Thomas H. Howard ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Sickle Cell Disease ◽  
Blood Transfusion ◽  
Sickle Cell ◽  
Stroke Prevention ◽  
Case Series ◽  
Acute Chest Syndrome ◽  
Cell Disease ◽  
Transfusion Therapy ◽  
Indirect Revascularization

Abstract Introduction Regular blood transfusion therapy is the standard care for secondary prevention of strokes in sickle cell disease (SCD). Despite regular blood transfusion therapy approximately 45% of the children with strokes will have progressive neurological disease (overt strokes or new silent cerebral infarcts) with an incidence of overt strokes of 3.2 events/100 patient-years (95% confidence interval, 1.3-6.5) (Hulbert, Blood 2011). Limited additional therapeutic options exist for these patients. Encephaloduroarteriosynangiosis (EDAS) is a neurosurgical procedure to improve cerebral blood flow by transposing scalp arteries onto the surface of the brain. Five previously published series reported a total of 41 EDAS or indirect revascularization procedures on patients with SCD aged 3-22 yrs. Three of 41 patients (7%) had a stroke at 24 hours, 5 days and three weeks following EDAS/indirect revascularization. Additionally, one patient developed TIA 12 months later, two patients developed intracranial hemorrhage, and one patient died from a pulmonary embolus during an episode of acute chest syndrome 48 months post-EDAS. To date, the incidence of complications and efficacy of EDAS procedure in stroke prevention has not been well established. Objectives To examine the incidence of overt stroke pre and post-EDAS for patients on chronic transfusion. Methods We studied a pediatric cohort with history of HbSS and SB0 thalassemia on chronic transfusion for CNS injury who underwent EDAS at the University of Alabama at Birmingham. The incidence of overt stroke pre- and post-EDAS was reviewed. All pre-transfusion hemoglobin and percent Hemoglobin S levels were recorded from the time of their first recorded abnormal MRI. To determine the acute complications of EDAS, we reviewed the peri-operative hospital records at the time of EDAS, post-EDAS emergency room visits and chronic transfusion clinic visits post EDAS. Results A total of 13 patients on chronic transfusion for secondary stroke prevention underwent 17 EDAS procedures for recurrent stroke, progressive vascular disease, or neurologic change including psychosis and decline in neuropsychometric scores. The mean time to EDAS was 80 months (median 56) from their first abnormal MRI in the medical records. The patients’ mean pre-transfusion hemoglobin level was 9.4 g/dL and mean HbS was 29.5%. All participants (n=13) maintained a mean pre-transfusion HbS < 40%; 62% (8/13) participants maintained a mean HbS <30% (two patients with elevated HbS were transitioned to hydroxyurea as part of a clinical trial). Prior to EDAS, three patients had a new overt stroke during 81 patient years.(3.7 strokes per 100 pt yrs) One of 17 EDAS procedures was complicated by an acute stroke one month after the procedure. No additional strokes occurred in these patients during 34 patient years.(3 strokes per 100 pt yrs) One child developed a chronic subdural hematoma one month post-EDAS requiring burr hole drainage; this patient had a complete recovery. Conclusion This case series represents the largest cohort of EDAS procedures for children with SCD, and in combination with the literature, suggests that patients with progressive CNS disease may benefit from EDAS. A multicenter retrospective case series should be completed to identify risk factors for progression status post an EDAS procedure followed by a clinical trial to determine the effectiveness of the procedure versus regular blood transfusion therapy. Disclosures: No relevant conflicts of interest to declare.

Moyamoya Disease Predicts Progression of Cerebral Vasculopathy in Patients with Sickle Cell Disease Despite Chronic Transfusion Therapy

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 2071-2071 ◽  
Author(s):  
Alyssa M Schlenz ◽  
Michael U Antonucci ◽  
Rebecca Cafiero ◽  
Nina-Serena Burkett ◽  
Julie Kanter
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Moyamoya Disease ◽  
Stroke Prevention ◽  
Conflicts Of Interest ◽  
The Body ◽  
Cell Disease ◽  
Transfusion Therapy ◽  
Moya Moya Disease ◽  
History Of

Abstract Introduction: Patients with sickle cell disease (SCD) develop multi-organ complications due to hemolysis, inflammation, and vascular occlusion that results in small vessel obstruction throughout the body. In the brain, however, large cerebral vessels are also damaged resulting in occlusion or stenosis and subsequent development of abnormal collateral vasculature and moyamoya disease. Chronic red cell transfusion (CRCT) therapy significantly reduces the risk of stroke in children with abnormal transcranial doppler (TCD) studies and is also effective in reducing stroke recurrence in those with a history of overt or silent stroke; however, it is unclear if CRCT halts or reverses the progression of vasculopathy. The present study evaluated cerebrovascular stenosis and moya moya disease as risk factors for progression of vasculopathy over time in a cohort of patients with SCD who were started on CRCT therapy as children for stroke prevention. Methods: A retrospective cohort study (with IRB approval) was used to evaluate cerebrovascular changes in patients on CRCT.Patients were included in the study if they had received CRCT for stroke prevention for at least 12 months and had at least two serial magnetic resonance imaging and angiography (MRI/MRA) studies for review. For the imaging analysis, the patient's MRI/MRA closest to the initiation of CRCT (i.e. baseline imaging) was compared to the most recent imaging available by a neuro radiologist who was blind to the patient's clinical history. Additional demographic information included the patient's current age, gender, indication for CRCT, years on CRCT, and laboratory results for pre-transfusion % hemoglobin S (HbS). Results: Forty patients with SCD (current age: M = 16.48, SD = 5.10; 23 male, 17 female) were included. Average duration of CRCT therapy was 9.96 years (SD = 5.67) and average pre-transfusion HbS levels were 42.52% (SD = 9.88). Patients were initiated on therapy due to: overt stroke (n = 19), silent stroke (n = 2), and abnormal TCD (n = 20). Of the 20 patients initiated on therapy due to abnormal TCD, 7 were found to have abnormal MRI at baseline consistent with silent stroke. One of these patients was also found to have co-occurring moyamoya disease, despite no evidence of prior overt stroke. At baseline, 45% (n = 18) of patients had abnormal MRA and 25% (n = 10) had moyamoya disease. Progression of vasculopathy occurred in 15% (n = 6) of patients, all of whom had a history of moya moya disease at baseline (5 patients with overt stroke and 1 with silent stroke). Of the remaining 3 patients with moya moya disease at baseline, 2 remained stable with no improvement and 1 demonstrated improvement on MRA. For patients with abnormal MRA, but no history of moya moya disease (n = 9), 5 demonstrated improvement (2 patients with silent stroke and 3 with overt stroke). Conclusions: Progression of vasculopathy was common among patients with baseline moyamoya disease despite CRCT. Also notable, however, was improvement in vasculopathy (as defined by reduction of stenosis) in 6 patients, the majority of whom had not developed moyamoya prior to the initiation of CRCT suggesting that more mild vasculopathy can be reversed with early intervention. Patients with moya moya disease warrant ongoing annual assessment as they may require vascular bypass to prevent further worsening. Future large, multi-site investigations are needed to identify improved biomarkers and further understand characteristics of patients who demonstrate improvement versus progression of vasculopathy on CRCT. Disclosures No relevant conflicts of interest to declare.

Limitations of Clinical Trials in Sickle Cell Disease: A Case Study of the Multi-center Study of Hydroxyurea (MSH) Trial and the Stroke Prevention (STOP) Trial

Hematology ◽  
2007 ◽  
Vol 2007 (1) ◽  
pp. 482-488 ◽  
Author(s):  
Michael R. DeBaun ◽  
Joshua J. Field
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Stroke Prevention ◽  
Cell Disease ◽  
Transfusion Therapy ◽  
Stop Trial ◽  
History Of ◽  
Secondary Analyses ◽  
Multi Center Study ◽  
Center Study

AbstractIn the past two decades, two landmark randomized controlled trials (RCT) have been completed among individuals with sickle cell disease (SCD), the Multi-center Study of Hydroxyurea (MSH) trial and the Stroke Prevention (STOP) trial. The MSH trial tested the hypothesis that hydroxyurea will reduce the frequency of painful episodes for adults with hemoglobin SS who had a history of 3 or more painful episodes per year. The STOP trial tested the hypothesis that among children with hemoglobin SS and an elevated transcranial Doppler (TCD) velocity measurement, blood transfusion therapy would decrease the risk of an initial stroke. After completion, both trials have defined standard care for individuals with hemoglobin SS. The purpose of this review is to examine the limitations of the MSH and STOP trials. In the context of these trials, we will examine the effects of narrow inclusion criteria that primarily include participants with hemoglobin SS and secondary analyses that are prone to false-positive results. In addition, we describe how after publication of these two trials use of hydroxyurea and TCD assessment has drifted towards a standard practice without evidence of therapeutic efficacy among groups that were excluded from the trials. Finally, we suggest that rigorously conducted RCTs or at the minimum multicenter observation studies with strong methodology should be performed in these excluded subgroups to confirm a benefit of hydroxyurea or TCD measurement.

Severity of Iron Overload in Patients with Sickle Cell Disease Receiving Chronic Red Blood Cell Transfusion Therapy

Vox Sanguinis ◽  
2000 ◽  
Vol 79 (4) ◽  
pp. 265-265 ◽  
Author(s):  
C.P. Engelfriet ◽  
P. Harmatz ◽  
E. Butenski ◽  
K. Quirolo
Keyword(s):  
Sickle Cell Disease ◽  
Blood Cell ◽  
Iron Overload ◽  
Sickle Cell ◽  
Red Blood Cell ◽  
Red Blood Cell Transfusion ◽  
Cell Disease ◽  
Transfusion Therapy
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