First name
Christopher
Middle name
E
Last name
Dandoy

Title

Circulating endothelial cells and the study of vascular injury in children undergoing hematopoietic stem cell transplant.

Year of Publication

2022

Date Published

08/2022

ISSN Number

1592-8721

DOI

10.3324/haematol.2022.280788

Alternate Title

Haematologica

PMID

35979718

Title

The impact of donor type on outcomes and cost of allogeneic hematopoietic cell transplant for pediatric leukemia: a merged CIBMTR and PHIS analysis: Pediatric acute leukemia transplant risks and utilization.

Year of Publication

2020

Date Published

2020 May 25

ISSN Number

1523-6536

Abstract

<p><strong>IMPORTANCE: </strong>AlloHCT may be associated with significant morbidity and mortality that result in increased healthcare utilization. To date, no multi-center comparative cost analyses have been performed specifically evaluating alloHCT in children with acute leukemia.</p>

<p><strong>OBJECTIVES: </strong>To describe the relationship between survival and healthcare utilization while investigating the hypothesis that matched sibling donor (MSD) alloHCT has significantly lower inpatient healthcare utilization compared to unrelated donor (URD) and that among URD, umbilical cord blood transplants (UCB) will have higher initial but lower long-term utilization.</p>

<p><strong>DESIGN: </strong>Retrospective cohort study Setting: Clinical and transplant outcomes data from the Center for International Blood and Marrow Transplant Research (CIBMTR) were merged with inpatient cost data from the Pediatric Health Information System (PHIS) database using a probabilistic merge methodology.</p>

<p><strong>PARTICIPANTS: </strong>The merged dataset contained U.S. patients age 1-21 years who received alloHCT for acute leukemia from 2004-2011 with comprehensive CIBMTR data at a PHIS hospital.</p>

<p><strong>EXPOSURE: </strong>AlloHCT analyzed by donor type with specific analysis of utilization and costs using PHIS claims data.</p>

<p><strong>MAIN OUTCOME: </strong>The primary outcomes of overall survival (OS), leukemia free survival (LFS), and inpatient costs were evaluated using Kaplan-Meier curves, Cox, and Poisson models.</p>

<p><strong>RESULTS: </strong>632 patients were identified in both CIBMTR and PHIS. 5-year LFS was 60% for MSD, 47% for well-matched matched unrelated donor bone marrow (MUD), 48% for mismatched unrelated donor, and 45% for UCB (p=0.09). Total adjusted costs were significantly lower for MSD versus MUD by day 100 (adjusted cost ratio (ACR) 0.73, CI 0.62-0.86, p&lt;0.001), and higher for UCB versus MUD (ACR 1.27, CI 1.11-1.45, p&lt;0.001). By 2yrs, total adjusted costs remained significantly lower for MSD when compared to MUD (ACR 0.67, CI 0.56-0.81, p&lt;0.001) and higher for UCB compared to MUD (ACR 1.25, 95% CI 1.02-1.52, p=0.0280).</p>

<p><strong>CONCLUSIONS: </strong>UCB and MUD alloHCT provide similar survival outcomes; however, MUD alloHCT has a significant advantage in cost by day 100 and 2 years. Ongoing research is needed to determine if the cost difference among URD alloHCT remains significant with a larger sample size and/or beyond the 2 years following alloHCT.</p>

DOI

10.1016/j.bbmt.2020.05.016

Alternate Title

Biol. Blood Marrow Transplant.

PMID

32464284

Title

The natural history of BK polyomavirus and the host immune response after stem cell transplantation.

Year of Publication

2019

Date Published

2019 Dec 18

ISSN Number

1537-6591

Abstract

<p><strong>BACKGROUND: </strong>BK polyomavirus (BKPyV) is associated with symptomatic hemorrhagic cystitis after hematopoietic cell transplantation (HCT). Little is known about the host immune response, effectiveness of antiviral treatment, or impact of asymptomatic replication on long-term kidney function.</p>

<p><strong>METHODS: </strong>In children and young adults undergoing allogeneic HCT, we quantified BKPyV viruria and viremia (pre-HCT and at months 1-4, 8, 12, and 24 post-HCT) and tested associations of peak viremia ≥10 000 or viruria ≥109 copies/mL with estimated kidney function (eGFR) and overall survival for two years post-transplant. We examined factors associated with viral clearance by month 4 including BKPyV-specific T cells by enzyme-linked immune absorbent spot (ELISPOT) at month 3 and cidofovir use.</p>

<p><strong>RESULTS: </strong>We prospectively enrolled 193 participants (median age 10 years). 18% had viremia ≥10 000 copies/mL, and 45% had viruria ≥109 copies/mL in the first three months post-HCT. Among the 147 participants without cystitis (asymptomatic), 58 (40%) had any viremia. In the entire cohort and asymptomatic subset, viremia ≥10 000 copies/mL was associated with a lower creatinine/cystatin C eGFR two years post-HCT. Viremia ≥10 000 copies/mL was associated with a higher risk of death (adjusted hazard ratio, 2.2; 95% confidence interval, 1.1-4.2). Clearing viremia was associated with detectable BKPyV-specific T cells and viremia &lt;10 000 copies/mL, but not cidofovir exposure.</p>

<p><strong>CONCLUSIONS: </strong>Screening for BKPyV viremia after HCT identifies asymptomatic patients at risk for kidney disease and reduced survival. These data suggest potential changes to clinical practice, including prospective monitoring for BKPyV viremia to test virus-specific T cells to prevent or treat BKPyV replication.</p>

DOI

10.1093/cid/ciz1194

Alternate Title

Clin. Infect. Dis.

PMID

31851312

Title

Clinical risks and healthcare utilization of haematopoietic cell transplantation for sickle cell disease in the U.S. using merged databases.

Year of Publication

2017

Number of Pages

1823-1832

Date Published

2017 Nov

ISSN Number

1592-8721

Abstract

<p>Allogeneic hematopoietic cell transplant advances for sickle cell disease improve outcomes, but limited healthcare utilization analysis exists. We hypothesized that early transplant (age &lt; 10 years) improves outcomes and decreases healthcare utilization compared to late. We performed a retrospective study of US children transplanted for sickle cell during 2000-2013 using two large databases. Univariate and Cox models estimated associations between demographics, sickle cell severity, and transplant-related variables on mortality and chronic graft versus-host-disease; while, Wilcoxon, Kruskal-Wallis, or linear trend tests estimated against healthcare utilization. 161 patients with 90% 2-year overall survival (95% confidence interval [CI] 85-95%) displayed significantly higher mortality late versus early (hazard ratio (HR) 21, 95% CI 2.8-160.8, p=0.003) and unrelated compared to matched sibling donor (HR 5.9, 95% CI 1.7-20.2, p=0.005) and chronic graft versus host disease late (HR 1.9, 95% CI 1.0-3.5, p=0.034) and unrelated (HR 2.5, 95%CI 1.2-5.4; p=0.017). 176 merged patients had $467,747 median total adjusted transplant cost per patient (range: $344,029-$799,219). Matched sibling donor and low severity had the lowest healthcare utilization compared to other donor and severity types (p&lt;0.001 and p=0.022, respectively); late transplant demonstrated no association (p=0.775). Patients with 2-yr pre- and post-transplant data (N=41) showed significant admission (p&lt;0.001), length of stay (p&lt;0.001), and cost (p=0.008) reductions. Superior early transplant outcomes require prospective studies in young children without severe disease and an available matched sibling to provide conclusive evidence for this approach. Reduced post-transplant healthcare utilization inpatient care indicates that transplant may provide a sustained decrease in healthcare costs over time.</p>

DOI

10.3324/haematol.2017.169581

Alternate Title

Haematologica

PMID

28818869

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