Design of a cost–effectiveness analysis alongside a randomized trial of transplantation using umbilical cord blood versus HLA-haploidentical related bone marrow in advanced hematologic cancer

Roth, Joshua A.; Bensink, Mark; O’Donnell, Paul V.; Fuchs, Ephraim J.; Eapen, Mary; Ramsey, Scott D.

doi:10.2217/cer.13.95

Cited by 18 publications

(9 citation statements)

References 39 publications

(36 reference statements)

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“…Recently, utilization of haploidentical transplantation has been revived primarily due to the use of post‐transplantation cyclophosphamide (PTCy) as well as the development of novel methods of selective depletion of T‐cell subsets, such as the use of αβ TCD . Due to improved safety, ease in procurement, and perhaps lower costs, haploidentical donors are now considered to be a viable alternative donor source . In addition, HaploSCT offers several key advantages: almost all patients have a haploidentical first‐degree related donor from the family, the donors are readily available and highly motivated to donate, and the patients can proceed to transplant very quickly (in less than 3 weeks) .…”

Section: Introductionmentioning

confidence: 99%

An overview of conditioning regimens for haploidentical stem cell transplantation with post‐transplantation cyclophosphamide

2015

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Haploidentical related donors are an attractive alternative source of stem cells for allogeneic stem cell transplantation due to widespread availability and ease of stem cell procurement. Historically, haploidentical stem cell transplantation (HaploSCT) with extensive T-cell depletion has been associated with high rates of infectious complications and nonrelapse mortality (NRM). Post-transplantation cyclophosphamide (PTCy) has been shown to induce immune tolerance, effectively control graft-versus-host-disease (GVHD), and is associated with lower NRM, making it a preferred option for patients undergoing HaploSCT. Over the last decade, several groups investigated PTCy for GVHD prevention in HaploSCT; it is now successfully utilized with both myeloablative and nonmyeloablative conditioning regimens with survival comparable to HLAmatched transplantation. Future directions will focus on optimizing conditioning regimens by diagnosis, improving donor selection, and enhancing graft-versus-leukemia effect.

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Section: Introductionmentioning

confidence: 99%

An overview of conditioning regimens for haploidentical stem cell transplantation with post‐transplantation cyclophosphamide

2015

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“…Specific late effects preventive guidelines have been published for BMT survivors [32]. Other areas of BMT survivorship continue to be actively studied, particularly caregiver health [33][34][35], quality of life with GVHD [36][37][38], psychosocial burden [39][40][41], integration of care models [42,43], and health care utilization/financial toxicity [6,44,45]. To integrate aforementioned BMT survivorship issues and current preventive guidelines into clinical practice, a team effort, that can be facilitated by a dedicated LTFU clinic that follows BMT survivors lifelong, is required.…”

Section: Introductionmentioning

confidence: 99%

Lost in Transition: The Essential Need for Long-Term Follow-Up Clinic for Blood and Marrow Transplantation Survivors

Hashmi

Carpenter

Khera

et al. 2015

Biology of Blood and Marrow Transplantation

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Because of expanding indications and improvements in supportive care, the utilization of blood and marrow cell transplantation (BMT) to treat various conditions is increasing exponentially, and currently more than 60,000 BMTs are performed annually worldwide. By the year 2030, it is projected that the number of BMT survivors will increase 5-fold, potentially resulting in one half of a million survivors in the United States alone. As the majority of survivors now live beyond the first 2 years after BMT, they are prone to a unique set of complications and late effects. Until recently, BMT experts assumed responsibility for almost all of the care for these survivors, but now oncologists/hematologists, pediatricians, and internists are involved frequently in offering specialized care and preventive services to these survivors. To integrate and translate into clinical practice the unique BMT survivorship issues with current preventive guidelines, a team effort is required. This can be facilitated by a dedicated "long-term-follow-up (LTFU)" clinic that provides lifelong care for BMT survivors. In this review, we first illustrate with clinical vignettes the need for LTFU and then focus upon the following: (1) types of LTFU clinic models, (2) challenges and possible solutions to the establishment of LTFU clinic, and (3) vulnerable transition periods.

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“…However, economic analysis has rarely been performed alongside RCTs for HCT (with some exceptions regarding upcoming clinical trials 45 ), leaving an important gap in actionable information. Administrative claims data can fill that gap, but with important caveats.…”

Section: Discussionmentioning

confidence: 99%

Administrative Claims Data for Economic Analyses in Hematopoietic Cell Transplantation: Challenges and Opportunities

Preussler

Mau

Majhail

et al. 2016

Biology of Blood and Marrow Transplantation

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There is an increasing need for the development of approaches to measure quality, costs and resource utilization patterns among allogeneic hematopoietic cell transplant (HCT) patients. Administrative claims data provide an opportunity to examine service utilization and costs, particularly from the payer’s perspective. However, because administrative claims data are primarily designed for reimbursement purposes, challenges arise when using it for research. We use a case study with data derived from the 2007–2011 Truven Health MarketScan Research database to discuss opportunities and challenges for the use of administrative claims data to examine the costs and service utilization of allogeneic HCT and chemotherapy alone for patients with acute myeloid leukemia (AML). Starting with a cohort of 29,915 potentially eligible patients with a diagnosis of AML, we were able to identify 211 patients treated with HCT and 774 treated with chemotherapy only where we were sufficiently confident of the diagnosis and treatment path to allow analysis. Administrative claims data provide an avenue to meet the need for health care costs, resource utilization, and outcome information. However, when using these data, a balance between clinical knowledge and applied methods is critical to identifying a valid study cohort and accurate measures of costs and resource utilization.

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Design of a cost–effectiveness analysis alongside a randomized trial of transplantation using umbilical cord blood versus HLA-haploidentical related bone marrow in advanced hematologic cancer

Cited by 18 publications

References 39 publications

An overview of conditioning regimens for haploidentical stem cell transplantation with post‐transplantation cyclophosphamide

An overview of conditioning regimens for haploidentical stem cell transplantation with post‐transplantation cyclophosphamide

Lost in Transition: The Essential Need for Long-Term Follow-Up Clinic for Blood and Marrow Transplantation Survivors

Administrative Claims Data for Economic Analyses in Hematopoietic Cell Transplantation: Challenges and Opportunities

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