Variations in the Use of Transcranial Doppler and Chronic Transfusion Therapy for Stroke Prevention in Pediatric Populations at Sickle Cell Centers.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 1214-1214
Author(s):  
Andrew E. Mulberg ◽  
Martin Behm ◽  
Catherine Magia ◽  
Robert Adams ◽  
Richard Drachtman ◽  
...  
Keyword(s):  
High Risk ◽  
Sickle Cell ◽  
Transcranial Doppler ◽  
Stroke Prevention ◽  
Pediatric Patients ◽  
Pediatric Population ◽  
Transfusion Therapy ◽  
Pediatric Hematology ◽  
Blood Institute ◽  
American Society

Abstract The Stroke Prevention Trial in Sickle Cell Anemia (STOP I) demonstrated a 90% risk reduction of first stroke in high-risk pediatric patients with sickle cell disease (SCD) receiving chronic transfusion therapy (CTT) (Adams RJ et al. N Engl J Med1998;339:5–11). Transcranial Doppler (TCD) is key in assessing the need for CTT in children with SCD. A survey of the American Society of Pediatric Hematology/Oncology found wide variation in how hematologists are managing children with SCD (Lane PA et al. Blood2001;98:784a). Recent recommendations by the National Heart, Lung and Blood Institute (NHLBI) suggest TCD and CTT for stroke prevention in high-risk children with SCD; however, there are no data on the utilization of these guidelines in clinical practice. Therefore, we conducted a telephone survey of Sickle Cell Centers to assess the clinical use of TCD and CTT for stroke prevention in pediatric patients with SCD. Invitations to participate were mailed to Directors of 70 centers; interviews were conducted between December 7, 2005 and February 10, 2006. Results from 25 participating centers were analyzed for trends related to use of TCD and CTT in the care of children with SCD. Questions included frequency of TCD based on velocity: <170 cm/sec, 170–200 cm/sec, and >200 cm/sec (Table). Because some participants could not provide an accurate response, responses are not available for all 25 centers in some cases. Frequency of TCD Sickle Cell Centers N = 25 TCD <170 cm/sec Repeat every 6 mo 4 Repeat yearly 13 Repeat every 1–2 y 4 Repeat every 2 y 1 TCD 170–200 cm/sec Repeat every mo 7 Repeat every 2 mo 3 Repeat every 3 mo 6 Repeat every 4–5 mo 3 Repeat every 6 mo 6 TCD >200 cm/sec Repeat every mo 14 Repeat every 2 mo 1 Repeat every 3 mo 3 Based on clinical observations, participants estimated the percentage of patients with eventual worsening of TCD velocity to >200 cm/sec and the approximate number of months to reach that endpoint. Responses indicated an average of 23% and 11.3 months, respectively. Of the 25 centers, all but one indicated that patients with an abnormal TCD (>200 cm/sec) were typically placed on CTT. Four participants stated that results from the STOP trial were the basis for their decision. Fifteen centers indicated that the youngest age they would initiate CTT was 1–2 years. Only 2 directors would consider CTT in children <1 year, and 3 would not initiate CTT until age 5. Although the NHLBI guidelines suggest a hemoglobin S (Hb S) target of <30%, only 5 directors stated that this was their primary factor in determining frequency of CTT. In terms of typical CTT duration, 68% said therapy was indefinite or lifelong, 20% said <1 year, 4% said 2 years, and 4% said until age 18. Although the number of participants was limited, responses indicate there is a relatively wide variation in the application of the NHLBI recommendations for TCD use and CTT in pediatric patients with SCD. The importance of these findings should be correlated both with a more extensive sample of SCD centers and the reported incidence of stroke in the pediatric population.

Transcranial Doppler Measures In Patients with Sickle Cell Disease at High Risk for Stroke and Receiving Hydroxyurea: The HyRetro Ancillary Study.

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 1620-1620 ◽  
Author(s):  
Thomas Adamkiewicz ◽  
Nadine Odo ◽  
Abdullah Kutlar ◽  
Janet L Kwiatkowski ◽  
Robert J Adams
Keyword(s):  
High Risk ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Transcranial Doppler ◽  
Stroke Prevention ◽  
Cell Disease ◽  
Hemoglobin F ◽  
Transfusion Therapy ◽  
Ancillary Study ◽  
Increased Risk

Abstract Abstract 1620 Children with sickle cell disease (SCD) and increased Transcranial Doppler (TCD) sonography velocity measures are at increased risk for stroke. Although chronic transfusion decreases this risk ten fold, this form of therapy is burdensome, and includes risk of iron overload. Although it has been established that the risk of stroke is still present even after 30 months of transfusion therapy, the total length of transfusion therapy required is not known. Hydroxyurea (HU) therapy is effective in preventing SCD complication, although its effect in preventing SCD central nervous system complications is less clear and a matter of current investigation. During one of the clinical trials (STOP2), an ancillary study was conducted to examine TCD velocities in 76 patients with SCD receiving hydroxyurea (HU) for a variety of indications. TCD measures were defined as the highest time-averaged maximum mean velocity of one of 8 measures (from the following cerebral arteries: LMCAVM, LM1VM, LBIFVM, LDICAVM, RMCAVM, RM1VM, RBIFVM and RDICAVM), as per STOP protocol. Results from 10 patients at high risk for stroke (defined as one or more TCD >=200 cm/sec) were evaluated for the present analysis. Average age at start of HU was 10.7 +/− 3.2 standard deviation (SD) years, 6 were female. Eight did not receiving chronic transfusion in STOP (randomized observation arm STOP n=4; observed non randomized in STOP n=4), and 2 were on transfusion (randomized transfusion arm STOP n=1; STOP/STOP2 with cross over n=1). Reasons for HU therapy included primary stroke prevention (n=6); secondary stroke prevention (TIA n=1; overt ischemic stroke n=1), vaso-occlusive or acute chest episodes (n=2). Averaged HU dose (available in 8) was 15 +/− 3 SD mg/kg. Averaged measures, off and on HU for each patient, were used to calculate means. Averaged hematological indices on treatment were as follows: white blood cell count 10.2 +/− 1.8 SD × 10^3/mm^3, hemoglobin 8.1 +/− 0.7 gm/dL; Mean Corpuscular Volume 105.6 +/− 6.7 cu μm; reticulocyte count 11.1 +/− 2.3 %. Averaged hemoglobin F, available in 8, was 16.1 +/− 5.7 %. Results are in table: Table Groups Treatment Months TCDs (n) cm/sec Not transfused in STOP (n=8) Pre HU 44+/−23 5.3+/−2.5 203+/−28 HU 33+/−24 5.0+/−3.6 179+/−48* Transfused in STOP (n=2) Pre HU 64+/−19 10.5+/−0.7 160+/−0.5 HU 19+/−7 4.5+/−0.7 180+/−1.1 * Wilcoxon signed-rank test p=0.008 One patient receiving HU for secondary stoke prevention suffered an overt stroke. This patient had a first overt stroke 24 months prior to start of HU therapy. MRI showed right frontal watershed and bilateral lacunar infarcts. Severe stenosis of the left MCA was noted. Patient had a repeat stroke 13 months after start of HU. MRI showed ischemia with watershed distribution of the left frontal lobe, stenosis of A2 and occlusion of A1 segments. Three TCD before and two after start of HU were all > 220 cm/sec. Overall, decreasing TCD velocities were noted in 60% prior to HU (STOP transfusion, n=2) and in 50% on HU (STOP transfusion, n=0). In conclusion, TCD velocities decreased significantly in high risk patients receiving HU, that were not transfused in STOP. However, these results require cautious interpretation, as numbers of patients are small, and length of observation varied. Patients with very high TCD measures remain at risk. Further studies may elucidate if there is a role for HU in patients with abnormal TCDs. Disclosures: No relevant conflicts of interest to declare.

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 Encephaloduroarteriosynangiosis and encephalomyoarteriosynangiosis for treatment of moyamoya syndrome in pediatric patients with sickle cell disease

2015 ◽  
Vol 16 (1) ◽  
pp. 64-73 ◽  
Author(s):  
Christoph J. Griessenauer ◽  
Jeffrey D. Lebensburger ◽  
Michelle H. Chua ◽  
Winfield S. Fisher ◽  
Lee Hilliard ◽  
...  
Keyword(s):  
Sickle Cell ◽  
Stroke Prevention ◽  
Pediatric Patients ◽  
Case Series ◽  
Secondary Stroke Prevention ◽  
Moyamoya Syndrome ◽  
Cell Disease ◽  
Transfusion Therapy ◽  
The Mean

OBJECT Pediatric patients with sickle cell disease (SCD) and moyamoya syndrome (MMS) are at significant risk for cerebrovascular accidents despite chronic transfusion therapy. Encephaloduroarteriosynangiosis (EDAS) and encephalomyoarteriosynangiosis (EMAS) are additional therapeutic options for these patients. To date, the incidence of complications after and efficacy of EDAS and EMAS in stroke prevention in this population have been described in several institutional case series reports, but no randomized prospective trials have been reported. METHODS The authors retrospectively reviewed the cases of all pediatric patients at the University of Alabama at Birmingham with a history of homozygous hemoglobin S (HbS) and sickle cell/β-thalassemia (SB0 thalassemia) and on chronic transfusion therapy, including 14 patients with MMS who underwent EDAS or EMAS. RESULTS Sixty-two patients with SCD and on chronic transfusion therapy were identified. After exclusion of patients on chronic transfusion therapy for indications other than stroke prevention, 48 patients (77.4%) remained. Of those patients, 14 (29.1%) underwent EDAS or EMAS. Nine (18.8%) and 25 (52.1%) patients were on chronic transfusion therapy for primary or secondary stroke prevention, respectively, but did not undergo EDAS or EMAS. The 14 patients with SCD and radiological evidence of MMS and on chronic transfusion therapy for primary or secondary stroke prevention underwent 21 EDAS or EMAS procedures for progressive vascular disease (92.9% of patients), stroke (71.4%), and/or seizure (7.1%). The mean (± SD) time from initiation of chronic transfusion therapy to EDAS or EMAS was 76.8 ± 58.8 months. Complications included 1 perioperative stroke, 1 symptomatic subdural hygroma, 1 postoperative seizure, and 1 case of intraoperative cerebral edema that required subsequent cranioplasty. Before EDAS or EMAS, the stroke rate was calculated to be 1 stroke per 7.8 patient-years. One additional stroke occurred during the follow-up period (mean follow-up time 33.7 ± 19.6 months), resulting in a post-EDAS/EMAS stroke rate of 1 stroke per 39.3 patient-years, a 5-fold reduction compared with that in the pre-EDAS/EMAS period. The patients’ mean pre-EDAS/EMAS HbS level of 29.5% ± 6.4% was comparable to the mean post-EDAS/EMAS HbS level of 25.5% ± 6.1% (p = 0.104). CONCLUSIONS The results of this retrospective case series in a large cohort of pediatric patients with SCD and MMS suggest that EDAS/EMAS provides a stroke-prevention benefit with an acceptably low morbidity rate. Given the combined experience with EDAS and EMAS for this indication at this and other institutions, a prospective clinical trial to assess their efficacy compared with that of chronic transfusion therapy alone is warranted.

Pial synangiosis for moyamoya syndrome in children with sickle cell anemia: a comprehensive review of reported cases

2014 ◽  
Vol 36 (1) ◽  
pp. E12 ◽  
Author(s):  
Benjamin C. Kennedy ◽  
Michael M. McDowell ◽  
Peter H. Yang ◽  
Caroline M. Wilson ◽  
Sida Li ◽  
...  
Keyword(s):  
Sickle Cell ◽  
Brain Ischemia ◽  
Sickle Cell Anemia ◽  
Transcranial Doppler ◽  
Pediatric Patients ◽  
Significant Risk ◽  
Moyamoya Syndrome ◽  
Transfusion Therapy ◽  
Comprehensive Review

Object Pediatric patients with sickle cell anemia (SCA) carry a significant risk of developing moyamoya syndrome (MMS) and brain ischemia. The authors sought to review the safety and efficacy of pial synangiosis in the treatment of MMS in children with SCA by performing a comprehensive review of all previously reported cases in the literature. Methods The authors retrospectively reviewed the clinical and radiographic records in 17 pediatric patients with SCA treated at the Morgan Stanley Children's Hospital of New York (MSCHONY) who developed radiological evidence of MMS and underwent pial synangiosis between 1996 and 2012. The authors then added any additional reported cases of pial synangiosis for this population in the literature for a combined analysis of clinical and radiographic outcomes. Results The combined data consisted of 48 pial synangiosis procedures performed in 30 patients. Of these, 27 patients (90%) presented with seizure, stroke, or transient ischemic attack, whereas 3 (10%) were referred after transcranial Doppler screening. At the time of surgery, the median age was 12 years. Thirteen patients (43%) suffered an ischemic stroke while on chronic transfusion therapy. Long-term follow-up imaging (MR angiography or catheter angiography) at a mean of 25 months postoperatively was available in 39 (81%) treated hemispheres. In 34 (87%) of those hemispheres there were demonstrable collateral vessels on imaging. There were 4 neurological events in 1590 cumulative months of follow-up, or 1 event per 33 patient-years. In the patients in whom complete data were available (MSCHONY series, n = 17), the postoperative stroke rate was reduced more than 6-fold from the preoperative rate (p = 0.0003). Conclusions Pial synangiosis in patients with SCA, MMS, and brain ischemia appears to be a safe and effective treatment option. Transcranial Doppler and/or MRI screening in asymptomatic patients with SCA is recommended for the diagnosis of MMS.

scholarly journals Effects of Chronic Transfusion Therapy on transcranial Doppler Ultrasonography Velocities in Children with Sickle Cell Anemia at Risk for Primary Stroke: Baseline Findings from the Twitch Trial

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 87-87 ◽  
Author(s):  
Hamayun Imran ◽  
Banu Aygun ◽  
Barry R. Davis ◽  
Sara L. Pressel ◽  
William Herbert Schultz ◽  
...  
Keyword(s):  
High Risk ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Transcranial Doppler ◽  
Doppler Ultrasonography ◽  
Study Entry ◽  
Red Cell ◽  
Transfusion Therapy ◽  
The Mean ◽  
Red Cell Transfusion

Abstract Introduction: Transcranial Doppler ultrasonography (TCD) is an established screening modality used to predict stroke in children with sickle cell anemia (SCA). Children with abnormal TCD velocities are at high risk for primary stroke. Based on STOP and STOP 2 data, indefinite chronic red cell transfusion therapy is recommended for children with SCA and abnormal TCD velocity, defined as a maximum time-averaged mean velocity (TAMV) ≥200 cm/sec. The aim of the current analysis was to evaluate the long term effects of transfusion therapy on TCD velocities, in a large cohort of children with SCA who received chronic red cell transfusion therapy for primary stroke prophylaxis. Methods: The TCD With Transfusions Changing to Hydroxyurea (TWiTCH) study is a phase III, randomized, controlled, multicenter non-inferiority trial comparing hydroxyurea to transfusions for primary stroke prevention in children with SCA and abnormal TCD velocities (ClinicalTrials.gov NCT01425307). Children with SCA and a history of abnormal TCD who received at least 12 months of transfusion therapy were eligible to participate. All subjects’ index TCD velocities (the TCD examination that prompted the start of chronic transfusion therapy) were reviewed centrally to confirm study eligibility. At enrollment, study TCD velocities were obtained using the SONARA/tek TCD Module by trained examiners and reviewed centrally. In addition, all children were evaluated with brain magnetic resonance angiography (MRA) scans, which were also reviewed centrally. Associations between demographic, clinical, radiological, and laboratory findings and index/enrollment TCD velocities were examined. Results: One hundred and thirty eight children with complete data were included in this analysis. Mean age for the entire cohort at enrollment was 9.8±2.8 years; 40% were male and 98.8% had HbSS. Mean age at the diagnosis of index TCD was 5.5±2 years. The mean duration of transfusion therapy was 4.3±2.4 years with 63% receiving simple transfusions, 30% receiving partial exchange transfusions, and 7% undergoing erythrocytapheresis. The mean pre-transfusion hemoglobin at study entry was 9.1±0.8 gm/dL and the mean pre-transfusion %HbS for the last 6 months prior to study entry was 29.5±8.3%. The average index TCD TAMV was 217±22 cm/sec (range 147-325). At study entry, the average TAMV was lower at 150±27cm/sec overall (142±27 cm/sec on the left and 140±29 cm/sec on the right). In 77% of the subjects, the TCD velocities had decreased to normal levels (<170 cm/sec), while they remained conditional in 21% and abnormal in 3%. The average decline from index to study entry TCD was 67±31 cm/sec. Nineteen subjects (13.7%) had severe vasculopathy on brain MRA. Higher recent pre-transfusion %HbS (p=.04), fewer years of transfusion therapy (p<.001), and the presence of severe vasculopathy (p<.001) were associated with higher TCD velocities at study entry and with larger declines in velocity from index to study entry. Higher index TCD values were also associated with larger declines in velocity from index to study entry (p<.001). Age and transfusion type did not have an effect on TCD values. Conclusions: Most of the children with SCA at high risk for primary stroke enrolled in the TWiTCH study have normal TCD velocities at study entry, although 21% have conditional and 3% still have abnormal velocities. Longer duration of transfusion therapy was associated with lower TCD velocities, while the presence of severe vasculopathy on brain MRA and recent higher pre-transfusion %HbS values were associated with higher TCD velocities. The TWiTCH trial will serially perform TCD examinations to compare the effects of hydroxyurea versus transfusions. Disclosures Off Label Use: Use of hydroxyurea in children with sickle cell disease.

Investigation of High-Risk ST131 Clone in Extended Spectrum β-Lactamase–Producing Escherichia coli Isolates in Children

2021 ◽  
Author(s):  
Mehmet E. Bulut ◽  
Gülen Hürkal ◽  
Nazan Dalgıç
Keyword(s):  
Escherichia Coli ◽  
High Risk ◽  
Pediatric Patients ◽  
Pediatric Population ◽  
Maldi Tof Ms ◽  
E Coli ◽  
Maldi Tof ◽  
Extended Spectrum ◽  
St131 Clone ◽  
Tof Ms

Abstract Objective Antimicrobial resistance poses a serious threat to children's health. In recent years, high-risk Escherichia coli ST131 has become an important target for global surveillance studies. The E.coli ST131 clone is associated with extended spectrum β-lactamase (ESBL) production, as well as multidrug resistance and treatment failure. Studies on this clone in the pediatric age group are limited. We aim to investigate the rate of high-risk E. coli ST131 clone in ESBL-positive E. coli isolates obtained from pediatric patients. Methods A total of 292 ESBL-positive E. coli isolates from clinical samples of pediatric patients was included in the study. MALDI-TOF MS system was used for bacterial identification. Susceptibility tests were performed using BD Phoenix automated system. ST131 detection was done by MALDI-TOF-MS. Fisher's exact test was used to compare the groups (significance <0.05). Results A total of 292 isolates was analyzed. The high-risk ST131 clone was detected in 117 (40%) of the 292 ESBL-positive isolates. ST131 rates were found to be significantly higher in children under the age of 5 years compared with children over the age of 5 years (49.3 vs. 31.1%, p = 0.0019). Ciprofloxacin resistance was higher in ST131 isolates (45.6 vs. 31.7%; p < 0.05). Conclusion The rate of the ST131 clone was found to be high in the pediatric population. The significantly high rate of resistance to ciprofloxacin, which is not commonly used in the pediatric population, in ST131 isolates reveals the importance of the spread of high-risk clones for the development of resistance.

scholarly journals Ischemic Stroke in Children and Young Adults with Sickle Cell Disease (SCD) in the Post-STOP Era

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 68-68 ◽  
Author(s):  
Janet L. Kwiatkowski ◽  
Julie Kanter ◽  
Heather J. Fullerton ◽  
Jenifer Voeks ◽  
Ellen Debenham ◽  
...  
Keyword(s):  
Ischemic Stroke ◽  
High Risk ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Stroke Prevention ◽  
Cell Disease ◽  
Clinical Series ◽  
The Mean

Abstract Background: The Stroke Prevention Trial in Sickle Cell Anemia (STOP) and Optimizing Primary Stroke Prevention in Sickle Cell Anemia (STOP 2) established routine transcranial Doppler ultrasound (TCD) screening with indefinite chronic red cell transfusions (CRCT) for children with abnormal TCD as standard of care. To identify children at high-risk of stroke, annual TCD screening is recommended from ages 2 to 16 years, with more frequent monitoring if the result is not normal. A reduction in stroke incidence in children with SCD has been reported in several clinical series and analyses utilizing large hospital databases when comparing rates before and after the publication of the STOP study in 1998. We sought to determine the rate of first ischemic stroke in a multicenter cohort of children who had previously participated in the STOP and/or STOP 2 trials and to determine whether these strokes were screening or treatment failures. Subjects and Methods: Between 1995 and 2005, STOP and STOP 2 (STOP/2) were conducted at 26 sites in the US and Canada. These studies included 3,835 children, ages 2 to 16 y with SCD type SS or S-beta-0-thalassemia. Participation in STOP/2 ranged from a single screening TCD to randomization. STOP 2 also had an observational arm for children on CRCT for abnormal TCD whose TCD had not reverted to normal. The Post-STOP study was designed to follow-up the outcomes of children who participated in one or both trials. 19 of the 26 original study sites participated in Post-STOP, contributing a total of 3,539 (92%) of the STOP/2 subjects. After exit from STOP/2, these children received TCD screening and treatment according to local practices. Data abstractors visited each clinical site and obtained retrospective data from STOP/2 study exit to 2012-2014 (depending on site) including follow-up TCD and brain imaging results, clinical information, and laboratory results. Two vascular neurologists, blinded to STOP/2 status and prior TCD and neuroimaging results, reviewed source records to confirm all ischemic strokes, defined as a symptomatic cerebral infarction; discordant opinions were resolved through discussion. For the first Post-STOP ischemic stroke, prior TCD result and treatment history subsequently were analyzed. Results: Of the 3,539 subjects, follow-up data were available for 2,850 (81%). Twelve children who had a stroke during STOP or STOP2 were excluded from these analyses resulting in data on 2,838 subjects. The mean age at the start of Post-STOP was 10.5 y and mean duration of follow-up after exiting STOP/2 was 9.1 y. A total of 69 first ischemic strokes occurred in the Post-STOP observation period (incidence 0.27 per 100 pt years). The mean age at time of stroke was 14.4±6.2 (median 13.8, range 3.5-28.9) y. Twenty-five of the 69 patients (36%) had documented abnormal TCD (STOP/2 or Post-STOP) prior to the stroke; 15 (60%) were receiving CRCT and 9 (36%) were not (treatment data not available for 1 subject). Among the 44 subjects without documented abnormal TCD, 29 (66%) had not had TCD re-screen in the Post-STOP period prior to the event; 7 of these 29 (24%) were 16 y or older at the start of Post-STOP, which is beyond the recommended screening age. Four of the 44 (9%) patients had inadequate TCD in Post-STOP (1 to 10.7 y prior to event). Six (14%) had normal TCD more than a year before the event (1.2 - 4 y); all but one of these children were younger than 16 y at the time of that TCD. Only 5 (11%) had a documented normal TCD less than 1 year prior to the event. Conclusions: In the Post-STOP era, the rate of first ischemic stroke was substantially lower than that reported in the Cooperative Study of Sickle Cell Disease, prior to implementation of TCD screening. Many (39%) of the Post-STOP ischemic strokes were associated with a failure to re-screen according to current guidelines, while only 11% occurred in children who had had recent low-risk TCD. Among those known to be at high risk prior to stroke, treatment refusal or inadequate treatment may have contributed. While TCD screening and treatment are effective at reducing ischemic stroke in clinical practice, significant gaps in screening and treatment, even at sites experienced in the STOP protocol, remain to be addressed. Closing these gaps should provide yet further reduction of ischemic stroke in SCD. Disclosures No relevant conflicts of interest to declare.

High risk of recurrent stroke after discontinuance of five to twelve years of transfusion therapy in patients with sickle cell disease

1991 ◽  
Vol 118 (3) ◽  
pp. 377-382 ◽  
Author(s):  
Winfred C. Wang ◽  
Edward H. Kovnar ◽  
Ina L. Tonkin ◽  
Raymond K. Mulhern ◽  
James W. Langston ◽  
...  
Keyword(s):  
High Risk ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Recurrent Stroke ◽  
Cell Disease ◽  
Transfusion Therapy

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.

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