Sickle cell disease: primum non nocere (first do no harm)

Montalembert, Mariane de; Roberts, Irene

doi:10.3324/haematol.2009.020172

Cited by 6 publications

(7 citation statements)

References 41 publications

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“…Some have cited this concept of nonmaleficence as a reason to potentially not perform HSCT in children with less severe SCD. 40 First, although the risk of transplantrelated mortality is low for HLA-identical sibling HSCT (,5%, possibly lower for children with less severe SCD 5 ), it is almost certainly higher than the short-term mortality risk of less severe SCD, as we now expect virtually all children with SCD in developed countries to survive childhood with current supportive care practices. 41,42 This small but important risk of death is, however, not the only potential harm of transplant.…”

Section: Is There Clinical Equipoise?mentioning

confidence: 99%

The ethics of a proposed study of hematopoietic stem cell transplant for children with “less severe” sickle cell disease

Nickel

Hendrickson

Haight

2014

Blood

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Hematopoietic stem cell transplant (HSCT) is the only cure for sickle cell disease (SCD). HSCT using an HLA-identical sibling donor is currently an acceptable treatment option for children with severe SCD, with expected HSCT survival >95% and event-free survival >85%. HSCT for children with less severe SCD (children who have not yet suffered overt disease complications or only had mild problems) is controversial. It is important to consider the ethical issues of a proposed study comparing HLA-identical sibling HSCT to best supportive care for children with less severe SCD. In evaluating the principles of nonmaleficence, respect for individual autonomy, and justice, we conclude that a study of HLA-identical sibling HSCT for all children with SCD, particularly hemoglobin SS and Sβ0-thalassemia disease, is ethically sound. Future work should explore the implementation of a large trial to help determine whether HSCT is a beneficial treatment of children with less severe SCD.

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Section: Is There Clinical Equipoise?mentioning

confidence: 99%

The ethics of a proposed study of hematopoietic stem cell transplant for children with “less severe” sickle cell disease

Nickel

Hendrickson

Haight

2014

Blood

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“…(21,31) At some European institutions, hematologists do not universally offer hydroxyurea to patients with SCA due to concerns about late and unknown effects. (12, 24) A recent qualitative study identified two distinct patterns of communication among American pediatric hematologists when discussing hydroxyurea with patients with SCA and their families. Bakshi et al describe a “collaborative approach” involving discussion of all treatment options with families versus a “proponent approach” defined by advocating for a pre-established treatment plan.…”

Section: Discussionmentioning

confidence: 99%

“…Physicians report not initiating hydroxyurea because of concerns about patient adherence. (21–24) Patients and families worry about side effects, often lack understanding of how hydroxyurea works, and cite insufficient discussion of their concerns as barriers to hydroxyurea initiation. (25, 26) However, treatment acceptance could be improved by discussing hydroxyurea with parents and patients in ways that define the indications for drug use, explain the potential benefits of therapy, acknowledge patient and family concerns, and reduce the burden of clinic attendance or obtaining medication refills.…”

mentioning

confidence: 99%

Targeted Hydroxyurea Education after an Emergency Department Visit Increases Hydroxyurea Use in Children with Sickle Cell Anemia

Pecker

Kappa

Greenfest

et al. 2018

The Journal of Pediatrics

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“…Hematopoietic stem cell transplantation (HSCT) is an important treatment option for children with severe and refractory disease and debilitating clinical complications [ 3 – 5 ]. Moreover, in the last 10 years, HSCT has been considered the only definitive curative treatment for severe SCD, with small mortality and morbidity rates [ 1 , 3 , 6 – 12 ]. Until 2014, about 500 SCD children had undergone HSCT in the world, resulting in a SCD-free survival rate of 95% [ 11 ].…”

Section: Introductionmentioning

confidence: 99%

“…Until 2014, about 500 SCD children had undergone HSCT in the world, resulting in a SCD-free survival rate of 95% [ 11 ]. The benefits of HSCT for SCD include family quality of life, stabilization, or restoration function in affected organs (central nervous system and lung) [ 6 , 7 ].…”

Section: Introductionmentioning

confidence: 99%

A Child as a Donor for Hematopoietic Stem Cell Transplantation: Bioethical Justification—A Case Study on Sickle Cell Disease

Pereira

Hellman

Hamerschlak

et al. 2017

Case Reports in Hematology

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Hematopoietic stem cell transplantation (HSCT) is an important treatment option for children with severe and refractory sickle cell disease (SCD) with debilitating clinical complications. HSCT with cells from the bone marrow of a HLA-identical sibling used in SCD has a low mortality risk, high cure rate, and high event-free survival rate after a median follow-up of 5-6 years. However, matched donors are found in only about 20% of the patients. A boy aged 8 years with SCD had a sister, <2 years old, a fully compatible donor. The boy met all eligibility criteria to undergo HSCT, and he was suffering from cognitive and neurologic impairment due to ischemic events. A Bioethical Committee jointly discussed the ethical issues on this case after a pediatric evaluation released the very young sister for donation. The justification was that the sister would benefit from the donation too because of the greater likelihood of survival and cure and less suffering of her brother. The parents were informed about the risks and benefits for both children, and the family was psychologically evaluated. After their consent, HSCT was performed and the patient is cured from SCD. The complication for the donor was the need for blood transfusion.

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Sickle cell disease: primum non nocere (first do no harm)

Cited by 6 publications

References 41 publications

The ethics of a proposed study of hematopoietic stem cell transplant for children with “less severe” sickle cell disease

The ethics of a proposed study of hematopoietic stem cell transplant for children with “less severe” sickle cell disease

Targeted Hydroxyurea Education after an Emergency Department Visit Increases Hydroxyurea Use in Children with Sickle Cell Anemia

A Child as a Donor for Hematopoietic Stem Cell Transplantation: Bioethical Justification—A Case Study on Sickle Cell Disease

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