Stroke Prevention Trial in Sickle Cell Anemia (STOP): extended follow-up and final results

Lee, Margaret T.; Piomelli, Sergio; Granger, Suzanne; Miller, Scott T.; Harkness, Shannon; Brambilla, Donald; Adams, Robert J.

doi:10.1182/blood-2005-10-009506

Cited by 263 publications

(200 citation statements)

References 21 publications

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“…10 Chronic transfusion therapy is the mainstay treatment for primary and secondary neurovascular complications. 2,3,12,22,30,35 Although chronic transfusion therapy prevents initial and recurrent stroke in the majority of patients, some patients on chronic transfusion therapy continue to experience strokes and develop progressive vascular disease. 19,30 The progression of their vascular disease triggers the development of a fragile network of arteries that resembles MMD and is present in 30%-43% of patients with SCD and a history of stroke.…”

Section: Discussionmentioning

confidence: 99%

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

Griessenauer¹,

Lebensburger

Chua

et al. 2015

PED

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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.

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Section: Discussionmentioning

confidence: 99%

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

Griessenauer¹,

Lebensburger

Chua

et al. 2015

PED

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“…46 STOP II further confirmed the markedly lower risk of stroke in participants receiving blood transfusions. 47 Nevertheless, serious complications of chronic transfusion include iron overload and alloimmunization, and these risks must be considered in medical decision-making regarding treatment. The recent availability of oral iron chelation agents is predicted to make transfusion a more acceptable treatment option 48 , and genetic testing for RH has the potential to reduce alloimmunization.…”

Section: Transfusion Management In Sickle Cell Disease (Scd)mentioning

confidence: 99%

The Structure and Function of the Rh Antigen Complex

Westhoff

2007

Seminars in Hematology

137

106

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The Rh system is one of the most important and complex blood group systems because of the large number of antigens and the serious complications for the fetus of a woman sensitized by transfusion or pregnancy. Major advances in our understanding of the Rh system have occurred with the cloning of the genes and with functional evidence that the Rh blood group proteins belong to an ancient family of membrane proteins involved in ammonia transport.The arrangement and configuration of the genes at the RH locus promotes genetic exchange, generating new antigens. Importantly, RH genetic testing can now be applied to clinical transfusion medicine and prenatal practice. This includes testing for RHD zygosity, confirmation or resolution of D antigen status, and detection of altered RHD and RHCE genes in individuals at risk for producing antibodies to high incidence Rh antigens, particularly sickle cell disease patients. The Rh proteins form a core complex that is critical to the structure of the erythrocyte membrane, and may play a physiologically role in the sequestration of blood ammonia. The Rh family of proteins now includes non-erythroid Rh homologs present in many other tissues, and comparative genomics reveals Rh homologs in all domains of life.

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“…While the mutation in the Hb molecule may be protective from malaria, this genetic modification leads to anemia and associated maladaptive consequences, including an increase in the incidence of stroke. 32 The risk of stroke can be reduced by treating anemia with blood transfusions. 32 Thus, the concept of anemia of chronic disease being an adaptive process requires further development.…”

Section: Is Anemia Of Chronic Disease An Adaptive Response?mentioning

confidence: 99%

“…32 The risk of stroke can be reduced by treating anemia with blood transfusions. 32 Thus, the concept of anemia of chronic disease being an adaptive process requires further development.…”

Section: Is Anemia Of Chronic Disease An Adaptive Response?mentioning

confidence: 99%

“…Quoique la mutation qui survient dans la molécule d'hémoglobine pourrait avoir un effet protecteur contre le paludisme, cette modification génétique provoque de l'anémie et des conséquences 'mal adaptatives' associées, y compris une augmentation de l'incidence d'AVC. 32 Le risque d'AVC peut être réduit en traitant l'anémie à l'aide de transfusions sanguines. 32 Dès lors, le concept selon lequel l'anémie des maladies chroniques est un processus adaptatif nécessite d'être approfondi.…”

Section: Les Changements De Seuils De Transfusionunclassified

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Prise en charge périopératoire de l’anémie aiguë et chronique: le retour du balancier est-il allé trop loin?

Hare

Baker

Mazer

2009

Can J Anesth/J Can Anesth

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Stroke Prevention Trial in Sickle Cell Anemia (STOP): extended follow-up and final results

Cited by 263 publications

References 21 publications

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

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

The Structure and Function of the Rh Antigen Complex

Prise en charge périopératoire de l’anémie aiguë et chronique: le retour du balancier est-il allé trop loin?

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