Debating the Future of Sickle Cell Disease Curative Therapy: Haploidentical Hematopoietic Stem Cell Transplantation vs. Gene Therapy

Kassim, Adetola A.; Leonard, Alexis

doi:10.3390/jcm11164775

Cited by 5 publications

(4 citation statements)

References 101 publications

(145 reference statements)

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“…Understanding super-utilizers based on health care encounters and expenditure is especially important when access to new medications and curative therapy is limited 32 , 33 and the cost-effectiveness of these treatments is unknown. Novel therapies such as transplant and gene therapy have the potential to lower expenditures, especially if pursued by those who are super-utilizers with high rates of vaso-occlusive pain episodes, because data suggest these can reduce vaso-occlusive pain episodes that require short-term health care use.…”

Section: Discussionmentioning

confidence: 99%

Comparing super-utilizers and lower-utilizers among commercial- and Medicare-insured adults with sickle cell disease

MacEwan,

Chiang,

O’Brien

et al. 2023

Blood Advances

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Sickle cell disease (SCD) is a rare but costly condition in the US. Super-utilizers have been defined as a subset of the population with high healthcare encounters or expenditures. While super-utilizers have been described in other disease states, little is known about super-utilizers among adults with SCD. This study aimed to characterize differences in expenditures, overall healthcare encounters, and pain episode encounters between super-utilizers (top 10% expenditures) and lower-utilizers with SCD (high: top 10-24.9%, moderate: 25-49.9%, and low: bottom 50% expenditures). A retrospective longitudinal cohort of adults with SCD were identified using validated algorithms in MarketScan and Medicare claims databases from 2016-2020. Encounters and expenditures were analyzed from inpatient, outpatient, and emergency department settings. Differences in encounters and expenditures between lower- and super-utilizers were compared with logistic regression. Among super-utilizers, differences in encounters and expenditures were compared according to incidence of pain episode encounters. The study population included 5,666 commercial-insured and 8,600 Medicare-insured patients. Adjusted total annual healthcare expenditure was 43.46 times higher for super-utilizers as compared to low-utilizers among commercial-insured and 13.37 times higher in Medicare-insured patients. Among super-utilizers, there were patients with few pain episode encounters who had higher outpatient expenditures compared to patients with a high number of pain episode encounters. Our findings demonstrate the contribution of expensive outpatient care among SCD super-utilizers, where analyses of high expenditure have largely focused on acute care. Future studies are needed to better understand super-utilizers in the SCD population to inform the effective use of preventative interventions and/or curative therapies.

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Section: Discussionmentioning

confidence: 99%

Comparing super-utilizers and lower-utilizers among commercial- and Medicare-insured adults with sickle cell disease

MacEwan,

Chiang,

O’Brien

et al. 2023

Blood Advances

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“…9 Potentially curative therapies such as stem cell transplantation and gene therapy are fascinating but reducing toxicities and costs will be important to allow wider accessibility. 10…”

Section: Sickle Cell Diseasementioning

confidence: 99%

Stroke in Children: Key Advances in the Field and the Next 20 Years

Kirton,

Jordan

2024

Stroke

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Cerebral arteriopathy, a frequent cause of stroke in children, and its relation to infection have been an active area of research with 2 large observational studies completed (VIPS I and II). 11,12 Two multicenter clinical trials are in progress that aim to determine if high-dose methylprednisolone in children with focal cerebral arteriopathy can improve or stabilize disease and reduce arterial ischemic stroke recurrence (the PASTA trial [Pediatric Arteriopathy Steroid Aspirin] and the FOCAS trial [Focal Cerebral Arteriopathy Steroid]). 13,14 The genetics of more diverse arteriopathies are not well understood but rapid progress suggests these will be important targets in the coming decades.

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“…HSCT complications can exacerbate SCD-related organ damage, emphasizing the importance of careful recipient and donor selection, appropriate conditioning intensity, and effective GvHD prophylaxis. By addressing these factors, the aim is to minimize morbidity and mortality associated with HSCT and improve patient outcomes 15 , 19 , 20 .…”

Section: Allogeneic Hsct In Scdmentioning

confidence: 99%

Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: A Multidimentional Review

Rostami,

Rad,

Rostami

et al. 2024

Cell Transplant

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While exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia) have been approved by the US Food and Drug Administration (FDA) as the first cell-based gene therapies for the treatment of patients 12 years of age and older with sickle cell disease (SCD), this treatment is not universally accessible. Allogeneic hematopoietic stem cell transplant (HSCT) has the potential to eradicate the symptoms of patients with SCD, but a significant obstacle in HSCT for SCD is the availability of suitable donors, particularly human leukocyte antigen (HLA)-matched related donors. Furthermore, individuals with SCD face an elevated risk of complications during stem cell transplantation due to SCD-related tissue damage, endothelial activation, and inflammation. Therefore, it is imperative to consider optimal conditioning regimens and investigate HSCT from alternative donors. This review encompasses information on the use of HSCT in patients with SCD, including the indications for HSCT, conditioning regimens, alternative donors, and posttransplant outcomes.

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Debating the Future of Sickle Cell Disease Curative Therapy: Haploidentical Hematopoietic Stem Cell Transplantation vs. Gene Therapy

Cited by 5 publications

References 101 publications

Comparing super-utilizers and lower-utilizers among commercial- and Medicare-insured adults with sickle cell disease

Comparing super-utilizers and lower-utilizers among commercial- and Medicare-insured adults with sickle cell disease

Stroke in Children: Key Advances in the Field and the Next 20 Years

Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: A Multidimentional Review

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