Hematopoietic stem cell transplantation for sickle cell disease: Progress and challenges

Hulbert, Monica L.; Shenoy, Shalini

doi:10.1002/pbc.27263

Cited by 36 publications

(34 citation statements)

References 100 publications

(219 reference statements)

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“…While SCD is a disease with high symptom burden already, chemotherapy and prolonged hospitalization are difficult to prepare a family for. This often leads to higher levels of uncertainty for both the child and caregiver, and a potentially harder time adjusting to the experience of these new, intense side-effects (Hulbert and Shenoy, 2018). This is apparent in higher distress scores at baseline (compared with Child 3), and may explain markedly higher scores mid-treatment when symptom distress is particularly difficult.…”

Section: Discussionmentioning

confidence: 99%

Active Music Engagement and Cortisol as an Acute Stress Biomarker in Young Hematopoietic Stem Cell Transplant Patients and Caregivers: Results of a Single Case Design Pilot Study

Holochwost¹,

Robb

Henley

et al. 2020

Front. Psychol.

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This paper reports the results of a single case design pilot study of a music therapy intervention [the Active Music Engagement (AME)] for young children (age 3.51 to 4.53 years) undergoing hematopoietic stem cell transplantation (HCST) and their caregivers. The primary aims of the study were to determine feasibility/acceptability of the AME intervention protocol and data collection in the context of HCST. Secondary aims were to examine caregivers' perceptions of the benefit of AME and whether there were changes in child and caregiver cortisol levels relative to the AME intervention. Results indicated that the AME could be implemented in this context and that data could be collected, though the collection of salivary cortisol may constitute an additional burden for families. Nevertheless, data that were collected suggest that families derive benefit from the AME, which underscores the need for devising innovative methods to understand the neurophysiological impacts of the AME.

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Section: Discussionmentioning

confidence: 99%

Active Music Engagement and Cortisol as an Acute Stress Biomarker in Young Hematopoietic Stem Cell Transplant Patients and Caregivers: Results of a Single Case Design Pilot Study

Holochwost¹,

Robb

Henley

et al. 2020

Front. Psychol.

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“…Hematopoietic stem cell transplantation (HSCT or HCT) is still emerging as a treatment for SCD (Khan & Roberts, 2019 Shenoy, 2013). Curing SCD is an important objective because this is the only treatment option that will prevent or stop the progression of the organ damage associated with the disease (Hulbert & Shenoy, 2018). Stem cells are cells that have the capacity to divide into multiple cells.…”

Section: Hematopoietic Stem Cell Transplantationmentioning

confidence: 99%

“…Patient selection in terms of the optimal age and timing for using HSCT for the treatment of SCD continues to be explored (Angelucci et al, 2014, Bhatia & Sheth, 2015. The current trend is for younger patients and those with severe disease to receive this treatment (Bhatia & Walters, 2008;Hulbert & Shenoy, 2018). HSCT use in adults, patients with advanced disea se and children with SCD who are asymptomatic or with mild disease, is a more challenging decision (Angelucci et al, 2014;Guilcher et al, 2018).…”

Section: Factors Associated With Improved Outcomes After Hsctmentioning

confidence: 99%

The Role of Hematopoietic Stem Cell Transplantation in the treatment of Sickle Cell Disease

Wishner

Taylor

Williams³

et al. 2020

ijmsci

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Abstract Sickle cell disease (SCD) affects millions of people around the world and is associated with significant morbidity and premature mortality. It is a chronic, life-long illness that affects virtually every tissue in the body, worsens over time, with varying degrees of morbidity in everyone with the disease. Before hematopoietic stem cell transplant (HCST), the mainstay of the management of SCD included early identification of the disease through newborn screening, infection prophylaxis with vaccinations and antibiotics, management of pain crises, blood transfusions, and hydroxyurea. These treatments although beneficial, do not cure SCD, stop the progressive end-organ damage associated with this disease, and are lifelong. Hematopoietic stem cell transplant is one of two treatment options that offer a cure for SCD and stops the progressive end-organ damage. The purpose of this article is to examine traditional treatments (best medical practice) and HCST for SCD and their associated complications. The role of HCST in the treatment of sickle cell disease, as well as recent research on HSCT as a cure for SCD, risk factors, patient selection, limitations, and future use of this treatment option, are also reviewed. Major issues surrounding the use of HCST for treating SCD include the optimal age for transplantation, disease severity, donor source, and the conditioning regimen before transplantation. The future of HCST including gene therapy is also discussed.

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“…At some centers, HSCT is now being offered to children with less severe disease 15 . However, the referral practice for HSCT is widely variable and not enough children with SCD are being offered HSCT 7,16,17 …”

Section: Introductionmentioning

confidence: 99%

Hematopoietic stem cell transplant referral patterns for children with sickle cell disease vary among pediatric hematologist/oncologists’ practice focus: A Sickle Cell Transplant Advocacy and Research Alliance (STAR) study

Meier

Abraham

Ngwube

et al. 2021

Pediatric Blood & Cancer

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Background: Hematopoietic stem cell transplantation (HSCT) provides a curative therapy for children severely affected by sickle cell disease (SCD). Rejection-free survival after matched sibling donor (MSD) HSCT is very high, but adoption of HSCT as a curative SCD therapy has been slow. In this study, we assess providers' perceptions about MSD HSCT for children with variable SCD severity, and determine the influence of provider characteristics on HSCT referrals. Procedure: After our Institutional Review Board deemed the study exempt, American Society of Pediatric Hematology/Oncology Clinical Forum listserv subscribers and American Society of Hematology members who self-identified as pediatric hematologists/oncologists (PHO) were emailed a survey. Analysis was performed to describe and evaluate correlations between participant demographics (including practice focus within PHO) and likelihood of HSCT referral for each scenario. Results: Spearman's rank correlation analysis did not reveal any significant relationship between demographic characteristics except practice focus and likelihood to refer to

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Hematopoietic stem cell transplantation for sickle cell disease: Progress and challenges

Cited by 36 publications

References 100 publications

Active Music Engagement and Cortisol as an Acute Stress Biomarker in Young Hematopoietic Stem Cell Transplant Patients and Caregivers: Results of a Single Case Design Pilot Study

Active Music Engagement and Cortisol as an Acute Stress Biomarker in Young Hematopoietic Stem Cell Transplant Patients and Caregivers: Results of a Single Case Design Pilot Study

The Role of Hematopoietic Stem Cell Transplantation in the treatment of Sickle Cell Disease

Hematopoietic stem cell transplant referral patterns for children with sickle cell disease vary among pediatric hematologist/oncologists’ practice focus: A Sickle Cell Transplant Advocacy and Research Alliance (STAR) study

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