Cognitive Dysfunction Occurs In Children with Sickle Cell Anemia without Cerebral Ischemic Insult or Vasculopathy

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 1076-1076
Author(s):  
Clarisse Lobo ◽  
Patricia Moura ◽  
Patricia Rio ◽  
Heber Maia ◽  
Juliana Fernandes ◽  
...  
Keyword(s):  
Academic Achievement ◽  
Cognitive Function ◽  
Cognitive Dysfunction ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Brain Mri ◽  
Neurocognitive Deficits ◽  
Ischemic Insult ◽  
Transfusion Therapy ◽  
Significant Difference

Abstract Abstract 1076 Background: Sickle cell anemia (SCA) has been associated with impairments in general cognitive functioning as measured by IQ scores and other neurocognitive measures. Children with SCA and overt stroke are the most affected, but those with silent infarcts (SI) on brain magnetic resonance imaging (MRI) also have cognitive impairment. Transcranial doppler ultrasonography (TCD) screening has allowed identification of individuals at high risk for stroke, in whom institution of prophylactic transfusion therapy produces a dramatic reduction in the incidence of primary stroke. Elevated TCD velocities have been reported to predict neurocognitive impairment in patients with SCA. We hypothesize that children with SCA with normal TCD velocities and no overt stroke or SI on brain MRI will have normal cognitive performance when compared with normal-matched controls. We investigated the academic achievement and cognitive function among children with SCA with normal brain MRI and normal TCD velocities, and compared their results with normal age and race matched controls. Methods: School age children with SCA who had normal MRI and TCD exams were evaluated. Controls (classmates and siblings) were matched for race, age, and social-economic status. SCA patients receiving chronic transfusion therapy or with a history of prior overt stroke were not eligible. All participants were followed at Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO). All patients and controls underwent MRI examination (to document the absence of stroke and SI) and cognitive testing. In addition, SCA subjects underwent TCD testing and had hematologic laboratory testing. SCA subjects were not experiencing pain or any other complication during study evaluations. The WISC-III was obtained as a measure of intellectual function, and achievement scores in reading, writing, and arithmetic were used to evaluate school performance. Comparisons among patients and controls were made using the Mann-Whitney test. This study was approved by the HEMORIO Ethics committee and all participants signed informed consent. Results: SCA patients had significantly lower total IQ and academic achievement than healthy controls (Table). Discussion: This is the first report showing cognitive dysfunction in children with SCA with both normal MRI and TCD examination. Early neurocognitive deficits are known to occur in children with SCA. In general, a patient with SCA in Brazil has a low socioeconomic status, low educational level, and poor access to health services, placing these children at higher risk of neurocognitive complications from the disease. Our study shows that children with SCA perform worse on measures of key cognitive function when compared with healthy matched controls, even without evidence of elevated velocities in the main cerebral arterial vessels or radiological insult to the brain. There was a significant difference in total IQ and school achievement among SCA patients and controls; however this was not true for verbal or executive IQ, suggesting that the cognitive compromise in this population is global. Cognitive dysfunction may occur long before lesions are observed on the MRI (ischemic insult) or on the TCD (vasculopathy), underscoring the need for preemptive screening and treatment of cognitive dysfunction in children with SCA. Disclosures: Off Label Use: Hydroxyurea for prevention of neuropsychological changes in SCD.

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.

Effects of Chronic Transfusion Therapy on MRI and MRA in Children with Sickle Cell Anemia at Risk for Primary Stroke: Baseline Imaging from the Twitch Trial

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 4052-4052 ◽  
Author(s):  
Kathleen J. Helton ◽  
Donna Roberts ◽  
William Herbert Schultz ◽  
Barry R. Davis ◽  
Theodosia A. Kalfa ◽  
...  
Keyword(s):  
Sickle Cell ◽  
Cerebral Artery ◽  
Sickle Cell Anemia ◽  
Internal Carotid ◽  
Brain Mri ◽  
Transfusion Therapy ◽  
Study Enrollment ◽  
Vessel Stenosis ◽  
History Of ◽  
The Mean

Abstract Introduction : Stroke is a major cause of morbidity and mortality in children with sickle cell anemia (SCA), and current efforts seek to prevent primary stroke in this high-risk population through transcranial Doppler (TCD) screening programs and prophylactic blood transfusions for children with abnormally high intracranial TCD velocities. During the SWITCH trial (ClinicalTrials.gov NCT00122980), a novel MRA vasculopathy grading (VOG) scale was developed, which documented baseline bilateral vessel stenosis and parenchymal injury in children receiving transfusions for secondary stroke prophylaxis, and provided a severity scale suitable for future clinical trials. We now describe the baseline brain magnetic resonance imaging/angiography (MRI/MRA) results and vasculopathy grading scores in children with SCA on primary stroke prophylaxis, and report correlations with intracranial TCD velocities and duration of transfusion. Methods: TCD With Transfusions Changing to Hydroxyurea (TWiTCH) is a Phase three randomized, controlled, multicenter clinical trial (ClinicalTrials.gov NCT01425307), comparing transfusions versus hydroxyurea for children with abnormal TCD velocities. Children were eligible for TWiTCH enrollment if they currently receive monthly transfusions for primary stroke prophylaxis, and had no known history of stroke, transient ischemic attack, or severe vasculopathy. All TWiTCH participants underwent baseline MRI/MRA examinations with central review after study enrollment. Baseline TCD examinations were also centrally reviewed and Time-Averaged Mean Velocities (TAMV) were recorded for the distal internal carotid artery (dICA), internal carotid bifurcation (BIF), first segment middle cerebral artery (MCA) = M1, mid MCA (MCA), and posterior cerebral artery (PCA). MRA vasculopathy grades were recorded bilaterally and compared to the TCD velocities and duration of transfusion. Results: Of 159 enrolled subjects, 143 had complete baseline MRI/MRA data. The mean age at study enrollment was 9.8 years, with a 2:3 male:female ratio, and mean age at the original abnormal (Index) TCD exam was 5.4 years. A total of 62 children had parenchymal abnormalities identified on baseline brain MRI, including 2 with infarcts, 54 with leukoencephalopathy, 5 with encephalomalacia, and 20 with lacunae lesions (not mutually exclusive). A total of 124 children had baseline MRA vasculopathy scores ≤3, while 19 had grades 4-6 (severe vasculopathy) that required removal from the study. There were significant positive associations between the baseline hemisphere vasculopathy grades and the corresponding TAMV velocities in the Left dICA (p=0.02), Left BIF (p˂.0001), Left MCA (p=.0002), Right BIF (p=.0003) and Right MCA (p=.0002). Likewise, when TCD velocities were categorized as low (<70 cm/s), normal (70-169 cm/s) or conditional/abnormal (≥170 cm/s), there were positive associations between left-sided vasculopathy and the left BIF (p=.04) and MCA (p=.003), along with right-sided vasculopathy with the right dICA (p=.04). The mean duration of transfusion for subjects with baseline MRA vasculopathy scores ≤3 was 1557 days, while those with severe vasculopathy (grades 4-6) was 1985 days; p= 0.053. Conclusions: Since the TWiTCH study excluded the enrollment of children on chronic transfusion therapy for primary stroke prevention with a known history of stroke, TIA, or severe vasculopathy, the prevalence of baseline brain MRI and MRA abnormalities was relatively higher than expected and 19 participants failed screening due to unrecognized severe vessel stenosis. The recent published SWiTCH vasculopathy grading scale was validated in the TWiTCH population, with more severe stenosis found to be associated with higher ipsilateral TCD velocities. Exit brain MRI/MRA exams will be compared to entry studies, to provide longitudinal data including comparisons between the effects of continued transfusions and hydroxyurea. Disclosures No relevant conflicts of interest to declare.

Hepatic Iron Overload in Children With Sickle Cell Anemia on Chronic Transfusion Therapy

2009 ◽  
Vol 31 (5) ◽  
pp. 309-312 ◽  
Author(s):  
Kathy Brown ◽  
Charu Subramony ◽  
Warren May ◽  
Gail Megason ◽  
Hua Liu ◽  
...  
Keyword(s):  
Iron Overload ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Hepatic Iron ◽  
Transfusion Therapy ◽  
Hepatic Iron Overload

Unexpected Anemia and Reticulocytopenia in an Adolescent With Sickle Cell Anemia Receiving Chronic Transfusion Therapy

2015 ◽  
Vol 37 (7) ◽  
pp. e438-e440
Author(s):  
Emily R. Blauel ◽  
Lily T. Grossmann ◽  
Madhav Vissa ◽  
Scott T. Miller
Keyword(s):  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Transfusion Therapy

STUDIES OF CONGENITAL HEMOLYTIC SYNDROMES

PEDIATRICS ◽  
1958 ◽  
Vol 22 (5) ◽  
pp. 910-922
Author(s):  
Marion E. Erlandson ◽  
Irving Schulman ◽  
Gertrude Stern ◽  
Carl H. Smith
Keyword(s):  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Fetal Hemoglobin ◽  
Severe Form ◽  
Intermediate Form ◽  
Transfusion Therapy ◽  
Effective Synthesis ◽  
Cooley's Anemia ◽  
Compensation Index

Rates of destruction of erythrocytes and of effective production of erythrocytes and hemoglobin have been determined in 10 patients with homozygous Cooley's anemia. The method employed was based upon survival of Cr51-labeled cells in patients in whom a state of equilibrium of erythrocytes was present. While a marked hemohytic defect is present, this defect does not, by itself, determine the degree of anemia present. Rates of effective production of erythrocytes are increased above normal but are not increased to the same degree found in patients with other hemolytic diseases. Rates of effective synthesis of hemoglobin were found to be less than those obtained for production of erythrocytes. The rates of production of fetal hemoglobin in these patients are remarkably elevated but cannot be directly correlated with the rate of destruction of erythrocytes, rate of production of erythrocytes, or the degree of anemia present. The hemolytic defect in patients with intermediate Cooley's anemia was comparable to that in the majority of the patients with the severe form of disease. However, the most marked hemolytic defects were among patients with the severe and not with the intermediate form of disease. Production of erythrocytes and hemoglobin did not differ significantly in the two forms of this disease. Results in two splenectomized patients did not differ significantly from results in the non-splenectomized group of patients. However, since pre-splenectomy data were not available, no statement may be made as to possible individual benefit derived from the operation. The final status of each patient is determined by the particular balance obtained between rates of destruction and production. Neither production nor destruction alone determines the degree of anemia. The compensation index, as a measure of final status in each patient, was lowest in the severe form of Cooley's anemia. It is presumed to be lower still in many patients who could not be studied because transfusion therapy was in progress. The compensation index is somewhat higher in patients with intermediate Cooley's anemia and in two splenectomized individuals not requiring frequent transfusions. Values in these patients approach the higher levels found in patients with sickle cell anemia and congenital spherocytosis.

scholarly journals Myth: Blood transfusion is effective for sickle cell anemia—associated priapism

CJEM ◽  
2006 ◽  
Vol 8 (02) ◽  
pp. 119-122 ◽  
Author(s):  
Andrew L. Merritt ◽  
Christopher Haiman ◽  
Sean O. Henderson
Keyword(s):  
Blood Transfusion ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Conventional Therapy ◽  
Case Reports ◽  
Complete Information ◽  
Effective Therapy ◽  
Transfusion Therapy ◽  
Medline Search ◽  
Neurologic Sequelae

ABSTRACTObjective:Priapism is a recognized complication of sickle cell anemia (SCA). When initial conventional treatments fail, simple or exchange blood transfusion has been advocated as a secondary intervention. However, recent literature suggests this may not be an effective therapy and may have significant neurologic sequelae. This paper reviews and summarizes the effectiveness and risks of blood transfusion compared with conventional priapism therapy.Methods:All relevant papers identified from a MEDLINE search were systematically examined for data related to the use of blood transfusion in the setting of priapism due to SCA. The effectiveness of conventional therapy was compared with transfusion therapy using the outcome of “time to detumescence” (TTD). In addition, papers documenting adverse neurologic sequela were reviewed and summarized.Results:Forty-two case reports were identified containing complete information with regard to patient age and TTD. The mean TTD was 8.0 days with conventional therapy (n= 16) and 10.8 days with blood transfusion therapy (n= 26). Adverse neurologic sequelae from blood transfusion therapy was described in 9 cases, with long term outcomes ranging from complete resolution to severe residual deficits.Conclusion:The current literature does not support the contention that blood transfusion is an effective therapy in the treatment of priapism due to SCA, as defined by an acceleration of TTD. In fact, numerous reports suggest that serious neurologic sequelae may result from this treatment. We feel the routine use of this therapy cannot be recommended.

scholarly journals Silent infarct is a risk factor for infarct recurrence in adults with sickle cell anemia

Neurology ◽  
2018 ◽  
Vol 91 (8) ◽  
pp. e781-e784 ◽  
Author(s):  
Lori C. Jordan ◽  
Adetola A. Kassim ◽  
Manus J. Donahue ◽  
Meher R. Juttukonda ◽  
Sumit Pruthi ◽  
...  
Keyword(s):  
Risk Factor ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Cox Regression ◽  
Brain Mri ◽  
Treatment Strategies ◽  
Significant Risk ◽  
Rank Test ◽  
Event Analysis ◽  
Log Rank Test

ObjectiveBecause of the high prevalence of silent cerebral infarcts (SCIs) in adults with sickle cell anemia (SCA) and lack of information to guide treatment strategies, we evaluated the risk of recurrent SCIs and overt stroke in adults with SCA with preexisting SCI.MethodsThis observational study included adults with SCA (HbSS or Sβ0 thalassemia) aged 18 to 40 years. Participants received 3-tesla brain MRI and a detailed neurologic examination. Time-to-event analysis assessed those with or without baseline SCI and with new or progressive infarcts. The incidence rate of new events was compared by log-rank test. Univariable Cox regression assessed the association of SCI with infarct progression.ResultsAmong adults with SCA with 2 MRIs and at least 6 months between MRIs (n = 54, mean interval = 2.5 years), 43% had SCI at baseline. Of participants with baseline SCI, 30% had new or progressive SCI over 2.5 years compared to 6% with no SCI at baseline; no participant had an overt stroke. New SCIs at follow-up were present in 12.9 per 100 patient-years with existing SCI compared with 2.4 per 100 patient-years without prior SCI (log-rank test, p = 0.021). No statistically significant differences were seen among those with or without baseline SCI in use of hydroxyurea therapy, hydroxyurea dose, or other stroke risk factors. The presence of SCI was associated with increased hazard of a new or progressive infarct (hazard ratio 5.27, 95% confidence interval 1.09–25.51, p = 0.039).ConclusionsSilent infarcts in adults with SCA are common and are a significant risk factor for future silent infarcts.

scholarly journals Hydroxyurea reduces cerebral metabolic stress in patients with sickle cell anemia

Blood ◽  
2019 ◽  
Vol 133 (22) ◽  
pp. 2436-2444 ◽  
Author(s):  
Melanie E. Fields ◽  
Kristin P. Guilliams ◽  
Dustin Ragan ◽  
Michael M. Binkley ◽  
Amy Mirro ◽  
...  
Keyword(s):  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Metabolic Stress ◽  
Brain Magnetic Resonance Imaging ◽  
Border Zone ◽  
Oxygen Extraction Fraction ◽  
Transfusion Therapy ◽  
Whole Brain ◽  
Disease Modifying Therapy ◽  
Disease Modifying

Abstract Chronic transfusion therapy (CTT) prevents stroke in selected patients with sickle cell anemia (SCA). We have shown that CTT mitigates signatures of cerebral metabolic stress, reflected by elevated oxygen extraction fraction (OEF), which likely drives stroke risk reduction. The region of highest OEF falls within the border zone, where cerebral blood flow (CBF) nadirs; OEF in this region was reduced after CTT. The neuroprotective efficacy of hydroxyurea (HU) remains unclear. To test our hypothesis that patients receiving HU therapy have lower cerebral metabolic stress compared with patients not receiving disease-modifying therapy, we prospectively obtained brain magnetic resonance imaging scans with voxel-wise measurements of CBF and OEF in 84 participants with SCA who were grouped by therapy: no disease-modifying therapy, HU, or CTT. There was no difference in whole-brain CBF among the 3 cohorts (P = .148). However, whole-brain OEF was significantly different (P &lt; .001): participants without disease-modifying therapy had the highest OEF (median 42.9% [interquartile range (IQR) 39.1%-49.1%]), followed by HU treatment (median 40.7% [IQR 34.9%-43.6%]), whereas CTT treatment had the lowest values (median 35.3% [IQR 32.2%-38.9%]). Moreover, the percentage of white matter at highest risk for ischemia, defined by OEF greater than 40% and 42.5%, was lower in the HU cohort compared with the untreated cohort (P = .025 and P = .034 respectively), but higher compared with the CTT cohort (P = .018 and P = .029 respectively). We conclude that HU may offer neuroprotection by mitigating cerebral metabolic stress in patients with SCA, but not to the same degree as CTT.

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