Risk of recurrent stroke in children with sickle cell disease receiving blood transfusion therapy for at least five years after initial stroke

Scothorn, Douglas J.; Price, Cynthia B.; Schwartz, Daniel L.; Terrill, Cindy; Buchanan, George R.; Shurney, Wanda; I, Sarniak; Fallon, Robert J.; Chu, Jhih‐Wei; Pegelow, Charles H.; Wang, Winfred C.; Casella, James F.; Resar, Linda S.; Berman, Brian; Adamkiewicz, Thomas V.; Hsu, Lewis L.; Ohene‐Frempong, Kwaku; Smith‐Whitley, Kim; Mahoney, Donald H.; Scott, John; Woods, Gerald M.; Watanabe, Masayo; DeBaun, Michael R.

doi:10.1067/mpd.2002.122498

Cited by 223 publications

(185 citation statements)

References 15 publications

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“…The need for secondary prevention in patients with SCD who have had a previous stroke is critical. 3,33 In a series studying 137 patients on chronic transfusion therapy for secondary stroke prevention, 31 patients had a second stroke while receiving chronic transfusion therapy. 33 Similar to the results described by Walters et al, 11 our series also showed a stabilizing effect on central nervous system function and prevention of future stroke.…”

Section: Discussionmentioning

confidence: 99%

“…3,33 In a series studying 137 patients on chronic transfusion therapy for secondary stroke prevention, 31 patients had a second stroke while receiving chronic transfusion therapy. 33 Similar to the results described by Walters et al, 11 our series also showed a stabilizing effect on central nervous system function and prevention of future stroke. However, neurological toxicities due to calcineurin inhibitors can occur following allo-SCT 34,35 as noted in one patient.…”

Section: Discussionmentioning

confidence: 99%

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Reduced toxicity, myeloablative conditioning with BU, fludarabine, alemtuzumab and SCT from sibling donors in children with sickle cell disease

Bhatia

Jin

Baker

et al. 2014

Bone Marrow Transplant

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BU and CY (BU/CY; 200 mg/kg) before HLA-matched sibling allo-SCT in children with sickle cell disease (SCD) is associated with 85% EFS but is limited by the acute and late effects of BU/CY myeloablative conditioning. Alternatives include reduced toxicity but more immunosuppressive conditioning. We investigated in a prospective single institutional study, the safety and efficacy of a reduced-toxicity conditioning (RTC) regimen of BU 12.8-16 mg/kg, fludarabine 180 mg/m 2 , alemtuzumab 54 mg/m 2 (BFA) before HLA-matched sibling donor transplantation in pediatric recipients with symptomatic SCD. Eighteen patients, median age 8.9 years (2.3-20.2), M/F 15/3, 15 sibling BM and 3 sibling cord blood (CB) were transplanted. Mean whole blood and erythroid donor chimerism was 91% and 88%, at days +100 and +365, respectively. Probability of grade II-IV acute GVHD was 17%. Two-year EFS and OS were both 100%. Neurological, pulmonary and cardiovascular function were stable or improved at 2 years. BFA RTC and HLA-matched sibling BM and CB allo-SCT in pediatric recipients result in excellent EFS, long-term donor chimerism, low incidence of GVHD and stable/improved organ function.Bone Marrow Transplantation (2014) 49, 913-920;

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Reduced toxicity, myeloablative conditioning with BU, fludarabine, alemtuzumab and SCT from sibling donors in children with sickle cell disease

Bhatia

Jin

Baker

et al. 2014

Bone Marrow Transplant

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“…Even with blood transfusion therapy, children with SCA and overt strokes still have a rate of infarct recurrence as high as 22% over a median of 10 years (overt stroke only) [78], or even 45% over 5.5 years (when both silent infarcts and overt stroke are included) [79]. Further after the first stroke while receiving blood transfusion therapy, 35% had a third stroke while receiving blood transfusion therapy with hemoglobin S levels as low as 1%, 9%, 22%, 25%, 26%, and 55% at the time of the second or third stroke [78,79].…”

Section: Stroke Prevention In Scamentioning

confidence: 99%

“…Further after the first stroke while receiving blood transfusion therapy, 35% had a third stroke while receiving blood transfusion therapy with hemoglobin S levels as low as 1%, 9%, 22%, 25%, 26%, and 55% at the time of the second or third stroke [78,79]. Based on the high rate of infarct recurrence even with rigorous blood transfsuion therapy, HLA-matched sibling hematopoietic stem cell transplantation is now considered the optimal therapeutic option for children with overt strokes.…”

Section: Stroke Prevention In Scamentioning

confidence: 99%

Evolution of sickle cell disease from a life‐threatening disease of children to a chronic disease of adults: The last 40 years

2015

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Over the past 40 years, public health measures such as universal newborn screening, penicillin prophylaxis, vaccinations, and hydroxyurea therapy have led to an impressive decline in sickle cell disease (SCD)-related childhood mortality and SCD-related morbidity in high-income countries. We remain cautiously optimistic that the next 40 years will be focused on meeting current challenges in SCD by addressing chronic complications of SCD to reduce mortality and improve quality of life in a growing population of adults with SCD in high-income countries, while simultaneously decreasing the disparity of medical care between high and low-income countries.

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“…Over a 4-year period, two studies 35,36 found high rates of stroke recurrence in children with SCD of nearly 30%.…”

Section: Stroke and Cardiac Diseasesmentioning

confidence: 99%

Acidentes vasculares encefálicos em pediatria

Filho¹,

Carvalho²

2009

J. Pediatr. (Rio J.)

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Objectives: To summarize 1) the definitions and epidemiological features of stroke in children; 2) the main risk factors that can lead to stroke in pediatrics and neonatology; 3) the main pathophysiological features involved in the genesis of brain injury in stroke; 4) the clinical manifestations and imaging diagnosis; and 5) the latest recommendations concerning the support measures, treatment, and prophylaxis of stroke in children.Sources: A review of the literature published in PubMed, EMBASE, and SciELO databases using the search terms stroke, pediatrics, and neonatology was performed, including relevant references from the chosen texts. Summary of the findings:Stroke in children are rare conditions, with incidence rates among two and eight per 100,000 children up to 14 years, and most cases show an underlying disease such as heart diseases, prothrombotic conditions, sickle-cell disease, and vascular malformations. There are no specific guidelines currently in place for the treatment of stroke in children, although central elements include support treatment, monitoring, and anticoagulation as secondary prevention in certain cases. Prognosis depends on the extent of brain damage and the underlying disease but recurrence rates are high in most cases. Conclusions:Early diagnosis of stroke in children is very important and pediatricians should be aware of the lack of specificity of the symptoms to avoid late sequelae and improve life quality.

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Risk of recurrent stroke in children with sickle cell disease receiving blood transfusion therapy for at least five years after initial stroke

Cited by 223 publications

References 15 publications

Reduced toxicity, myeloablative conditioning with BU, fludarabine, alemtuzumab and SCT from sibling donors in children with sickle cell disease

Reduced toxicity, myeloablative conditioning with BU, fludarabine, alemtuzumab and SCT from sibling donors in children with sickle cell disease

Evolution of sickle cell disease from a life‐threatening disease of children to a chronic disease of adults: The last 40 years

Acidentes vasculares encefálicos em pediatria

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