Chronic transfusion therapy effectiveness as primary stroke prophylaxis in sickle cell disease patients

Background The risk of neurological complications is increased in children with sickle cell disease (SCD), such as silent cerebral infarction (SCI) and stroke. Brain-Derived Neurotrophic Factor (BDNF) is a nerve growth factor associated with elevated transcranial Doppler (TCD) velocities and increased risk of stroke in SCD patients. So, we assessed the BDNF level in children with SCD and its relation to neurological complication as silent stroke. Methods A comparative cross-sectional study was conducted on 40 patients with SCD, recruited from the Hematology Unit, Pediatric Department, Menoufia University Hospital, and 40 healthy children as controls. Laboratory investigations including BDNF were done. TCD was done for all patients and Magnetic Resonance Imaging (MRI) was done on high-risk patients. Results BDNF levels were significantly higher in children with SCD than in controls with a significant relation to TCD findings. There was a statistically significant diagnostic ability of BDNF in the prediction of SCD complications as its sensitivity was 89.5%, specificity (95% CI) was 80% with a cut-off point >0.69, AUC = 0.702, and p = 0.004). Conclusion Serum BDNF levels were higher in sickle disease patients who had abnormal transcranial Doppler. BDNF had a significant diagnostic ability in the detection of SCD complications. Impact Silent stroke is a very serious complication in children with sickle cell disease, so regular follow up should be every six months. BDNF is considered a potential biomarker for stroke risk prediction in patients unable to receive TCD.

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“… 22 A Brazilian study reported 15 patients with abnormal TCD under chronic transfusion for primary prevention of stroke. 23 …”

Section: Discussionmentioning

confidence: 99%

Brain-derived neurotrophic factor and neuroimaging in pediatric patients with sickle cell disease

et al. 2023

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“…The iron overload approach was effective with oral chelator and only one patient had alloimmunization. 18 In this study, in the 18 children who had normalized TCD and no signs of vasculopathy or parenchymal brain injury, RTP was replaced by HU at MTD after one year. Before the publication of the TCD With Transfusions Changing to Hydroxyurea (TWiTCH) 19 study, French researchers already used the replacement of chronic transfusions by HU or HSCT as a stroke prevention protocol for patients with compatible donors after TCD normalization.…”

Section: Discussionmentioning

confidence: 91%

Follow-up of children with sickle cell anemia screened with transcranial Doppler and enrolled in a primary prevention program of ischemic stroke

Sabarense,

Silva,

Muniz

et al. 2024

Preprint

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Background: Stroke is a serious complication of sickle cell anemia (SCA). Transcranial Doppler (TCD) is the risk screening tool for ischemic strokes. Objective: The objective of the study was to describe the clinical progression of children with SCA who presented with high risk for stroke by TCD or relevant changes by magnetic resonance angiography (MRA) and underwent the regular transfusion program (RTP) and/or hydroxyurea (HU) treatment between 2007 and 2018. Method: This was a neonatal retrospective/prospective cohort study with children born between 1999 and 2014 with the homozygotic form (HbSS) or Sβ0-thalassemia, who underwent TCD at least once. Results: Of the 718 children screened during this period, 675 had HbSS and 43 HbSβ0-thalassemia. In 54 children (7.5%), all with HbSS, a high risk TCD (n=45) or, when the TCD was inconclusive, MRA with cerebral vasculopathy (n=9) was detected. Of these, 51 started RTP and the families of three refused treatment. Of the 43 children with high-risk TCD who initiated RTP, 29 (67.4%) reverted to low risk. In 18 of them (62%), HU was started at the maximum tolerated dose (MTD) before transfusion discontinuation. None of these 29 patients had a stroke. Eight children (18.6%) maintained a high-risk TCD even using RTP/HU, and two had a stroke. Conclusions: TCD was confirmed as a viable tool for tracking patients with a risk for stroke. The RTP was effective in preventing the primary event. New strategies are necessary to prevent stroke using HU and new drugs, besides bone marrow transplantation.

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“…Included studies are Ware et al [4], Bernaudin et al [22], Fridyland et al [25], DeBaun [11], DeBaun et al [34], Ware [35], Mulaku et al [36], Aygun et al [28], Aygun et al [29], Brousse et al [30], Mirre et al [31], Moreira Franco et al [32], Wood et al [33], Montalembert et al [37].…”

Section: Table 2: Axial Assessment Appraisal Results Of the Included ...mentioning

confidence: 99%

“…FIGURE 2: Forest plot summarizing the analysis of the effects of transfusion on stroke incidences in children with sickle cell anemia.Included studies are Aygun et al[29], Brousse et al[30], Mirre et al[31], Moreira Franco et al[32], and Wood et al[33].…”

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confidence: 99%

Blood Transfusion Vs. Hydroxyurea for Stroke Prevention in Children With Sickle Cell Anemia: A Systematic Review and Meta-Analysis

Hafiz¹,

Aldharman²,

AlSubaie³

et al. 2022

Cureus

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Sickle cell anemia (SCA) is a hereditary condition that can lead to severe complications in children such as acute coronary syndrome, splenic sequestration, renal failure, and stroke. Blood transfusion and hydroxyurea (HU) therapy are used to prevent stroke in children with sickle cell disease (SCD). Preliminary data show considerable variation and inconsistency in the use of these two therapeutic interventions. Therefore, this systematic review was carried out to compare the effects of blood transfusion to HU therapy in preventing stroke for children with sickle cell disease. There was an extensive literature search in reliable and authentic databases like PubMed, Medline, Scopus, Cochrane, and Science Direct to obtain relevant articles. This study used the standards and guidelines from the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA). During the systematic review, data were obtained focusing on the following parameters: the size of the sample in the study, the age of the subjects involved in the study, the type of Intervention, and the outcome. After an initial search of 163 papers, 25 studies were included. The results of the research give the first evidence that HU is effective in the treatment of cerebrovascular problems in children with SCD. However, it is unclear under what circumstances HU may prevent a second stroke. It can be concluded that children with SCA can effectively avoid primary strokes through chronic blood transfusions and HU.

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Chronic transfusion therapy effectiveness as primary stroke prophylaxis in sickle cell disease patients

Cited by 5 publications

References 19 publications

Brain-derived neurotrophic factor and neuroimaging in pediatric patients with sickle cell disease

Brain-derived neurotrophic factor and neuroimaging in pediatric patients with sickle cell disease

Follow-up of children with sickle cell anemia screened with transcranial Doppler and enrolled in a primary prevention program of ischemic stroke

Blood Transfusion Vs. Hydroxyurea for Stroke Prevention in Children With Sickle Cell Anemia: A Systematic Review and Meta-Analysis

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