Association of Matched Sibling Donor Hematopoietic Stem Cell Transplantation With Transcranial Doppler Velocities in Children With Sickle Cell Anemia

Bernaudin, Françoise; Verlhac, Suzanne; Latour, Régis Peffault de; Dalle, Jean‐Hugues; Brousse, Valentine; Petras, Eleonore; Thuret, Isabelle; Paillard, Catherine; Neven, Bénédicte; Garrigue, Claire; Divialle‐Doumdo, Lydia; Pondarré, Corinne; Guitton, Corinne; Missud, Florence; Runel, Camille; Jubert, Charlotte; Elana, Gisèle; Ducros-Miralles, Elisabeth; Drain, Élise; Taïeb, Olivier; Arnaud, Cécile; Kamdem, Annie; Malric, Aurore; Elmaleh‐Bergès, Monique; Vasile, Manuela; Leveillé, Emmanuella; Socié, Gérard; Chevret, Sylvie

doi:10.1001/jama.2018.20059

Cited by 61 publications

(64 citation statements)

References 40 publications

(54 reference statements)

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“…The DREPAGREFFE prospective trial comparing MSD-SCT to standard care in patients on long-term chronic transfusion for abnormal-TCD has been recently reported [80]. To be included, SCA-children had to be five to 15 years old, have at least one non-SCA sibling with parents agreeing to familial HLA-typing and SCT if available MSD.…”

Section: Who To Consider For Allogeneic Stem Cell Transplantation mentioning

confidence: 99%

“…Velocities were highly significantly lower in the transplantation than in the standard care group at 1- and 3-year follow-up (129.6 cm/s vs. 170.4 cm/s, difference −40.8 cm/s, p < 0.001 at 1 year and 112.4 cm/s vs. 156.7 cm/s, difference −44.3 cm/s, p < 0.001, at three years). This could be explained by the fact that with CT, patients have a double population of red cells with normal AA and SS, whereas after SCT, even in those transplanted with and AS donor, sickle cells are no longer present (Figure 1) [80]. Moreover, the proportion of patients with normalized velocities (<170 cm/s) was significantly higher after SCT than on standard-care (80% vs. 48% at 1 year; p = 0.045).…”

Section: Who To Consider For Allogeneic Stem Cell Transplantation mentioning

confidence: 99%

“…Children successfully engrafted after SCT had no new silent infarct occurrence on MRI [14,15,66,80]. Thus, SCT offers the best prevention for silent cerebral infarcts.…”

Section: Who To Consider For Allogeneic Stem Cell Transplantation mentioning

confidence: 99%

“…Following SCT, stabilization with age of cognitive performances has been found [100], but the DREPAGREFFE trial comparing chronic transfusion to HSCT in children with abnormal-TCD history did not find any significant difference in cognitive performances between both groups at the 1 and 3-year follow-up. However, a significantly higher progression of the processing speed index was seen in the transplantation group from 1 to 3 years : 6.1 (12.9) vs. −3.1 (10.5) in the standard-care group, p = 0.02 [80].…”

Section: Who To Consider For Allogeneic Stem Cell Transplantation mentioning

confidence: 99%

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Why, Who, When, and How? Rationale for Considering Allogeneic Stem Cell Transplantation in Children with Sickle Cell Disease

Bernaudin

2019

JCM

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Considering the progress made in the management of sickle cell disease during the past 30 years, along with the excellent results obtained with hematopoietic stem cell transplantation (SCT), it is important to reexamine why, who, when and how to recommend allogeneic SCT in children with sickle cell disease. While sickle cell disease has a low risk of death in children and a high risk for morbidity during aging, SCT carries an early risk of death, graft-vs-host disease and infertility. Nevertheless, SCT offers at least 95% chance of cure with low risk of chronic graft-vs-host disease when a matched-sibling donor is available and the risks of infertility can be reduced by ovarian, sperm or testis cryopreservation. Thus, all available therapies such as hydroxyurea, transfusions and SCT should be presented to the parents, providers, and affected children and discussed with them from infancy. Furthermore, the use of these therapies should be adjusted to the severity of the disease and to local availabilities in order to choose the treatment offering the best benefit/risk ratio.

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Section: Who To Consider For Allogeneic Stem Cell Transplantation mentioning

confidence: 99%

Section: Who To Consider For Allogeneic Stem Cell Transplantation mentioning

confidence: 99%

“…Children successfully engrafted after SCT had no new silent infarct occurrence on MRI [14,15,66,80]. Thus, SCT offers the best prevention for silent cerebral infarcts.…”

Section: Who To Consider For Allogeneic Stem Cell Transplantation mentioning

confidence: 99%

Section: Who To Consider For Allogeneic Stem Cell Transplantation mentioning

confidence: 99%

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Why, Who, When, and How? Rationale for Considering Allogeneic Stem Cell Transplantation in Children with Sickle Cell Disease

Bernaudin

2019

JCM

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“…Though progressive liver disease is not a currently recognized indication, efficacy has been demonstrated. [33][34][35][36]. SCT is likewise associated with the risk of graft versus host disease, as well as hepatic veno-oclusive disease [37].…”

Section: Discussionmentioning

confidence: 99%

Management of Sickle Cell Intrahepatic Cholestasis: An Argument in Favor of Automated Exchange Transfusion

Adkins¹,

Savani²,

Booth³

2019

CHI

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Sickle cell disease patients are commonly treated at transfusion medicine services, and understanding of the hepatic manifestations of the disease is key for optimal management, specifically, in individuals presenting with sickle cell intrahepatic cholestasis (SCIC). SCIC represents a rare, severe hepatic crisis wherein sinusoidal red cell sickling leads to massive hepatocyte dysfunction and cholestatic laboratory findings. Acute SCIC is defined by abdominal pain with progressive hepatic injury associated with hyperbilirubinemia, renal failure, encephalopathy, and coagulopathy. Patients are generally managed with red blood cell exchange transfusion (RBCEx), when available, as this is a potentially fatal condition. Simple transfusion may be utilized in resource-poor environment or when patients refuse RBCEx. As less than 50 adult cases have been described in the literature, many of them with limited follow-up, randomized clinical trials comparing RBCEx with other treatments are currently unfeasible. Likewise, a chronic form exists, but is less well characterized, and is associated with persistent bilirubinemia and a variable course in terms of progressive hepatic disease. We undertake a brief review of the literature and discuss two cases of SCIC managed with RBCEx at our institution.

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Accelerating the Science of SCD Therapies—Is a Cure Possible?

Benz

Mondoro

Gibbons

2019

JAMA

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Nearly 7 decades have passed since seminal studies of hemoglobin led to the characterization of sickle cell anemia as the first defined molecular disease. These foundational insights into sickle cell disease (SCD) initiated an exciting era of medicine that has substantially expanded the understanding of the molecular basis of thousands of disorders over the ensuing years. The field of molecular medicine is now at a pivotal moment in its history, a time when scientific capabilities to read, edit, and reprogram the human genetic code for therapeutic approaches are within reach. The rapid pace of innovation in emerging technologies of gene editing and translational research suggest that it is time to accelerate curative therapies for the first defined molecular disease.More than 100 years have passed since Archives of Internal Medicine (now JAMA Internal Medicine) published Herrick's description of misshapen red cells in a Grenadan medical student. 1 In hindsight, it is now understood how this"puzzling"clinicalobservationinanindividualofAfrican ancestry fits within the current "central dogma" of molecular medicine: DNA-RNA-protein-disease. Sickle cell disease is a hemolytic anemia caused by a mutation in the β globin subunit of adult hemoglobin (HbA) that substitutes valine for glutamic acid at position 6. When deoxygenated, HbS polymerizes, rendering the red cell rigid, viscous, and abnormallyadherenttothecapillaryendothelium.Thisimpedes blood flow in the microcirculation, causing ischemia and microinfarcts that lead to painful crises, strokes, renal failure, retinopathy, and myriad end-organ injuries.Although the discovery of the molecular basis of SCD was a triumphant milestone in molecular medicine, 2 it is still a tragic truth that in the absence of access to treatments, infants born with SCD are unlikely to live to adulthood in many parts of the world. Over the past 4 decades, advances such as penicillin prophylaxis, standardized vaccine schedules, transfusion support, and hydroxyurea therapy have significantly extended life expectancy for those with SCD. Nevertheless, many individuals with SCD in the United States still lack a patientcentered medical home with access to evidence-based care that optimizes health outcomes.Within the current therapeutic armamentarium, hydroxyurea is the first successful treatment strategy to address the molecular basis of SCD through the induction of a partial increase in the production of fetal hemoglobin (HbF). Fetal hemoglobin does not sickle and offers the added benefit of inhibiting deoxygenated-HbS polymerization. Approved by the US Food and Drug Administration (FDA) in the late 1980s, hydroxyurea prolongs patient survival and reduces painful crises and acute chest syndrome. Challenges and adverse effects

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Association of Matched Sibling Donor Hematopoietic Stem Cell Transplantation With Transcranial Doppler Velocities in Children With Sickle Cell Anemia

Cited by 61 publications

References 40 publications

Why, Who, When, and How? Rationale for Considering Allogeneic Stem Cell Transplantation in Children with Sickle Cell Disease

Why, Who, When, and How? Rationale for Considering Allogeneic Stem Cell Transplantation in Children with Sickle Cell Disease

Management of Sickle Cell Intrahepatic Cholestasis: An Argument in Favor of Automated Exchange Transfusion

Accelerating the Science of SCD Therapies—Is a Cure Possible?

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