Michael J. Eckrich

National Survey of Hematopoietic Cell Transplantation Center Personnel, Infrastructure, and Models of Care Delivery

Hematopoietic cell transplantation (HCT) is a complex procedure that requires availability of adequate infrastructure, personnel, and resources at transplantation centers. We conducted a national survey of transplantation centers in the United States to obtain data on their personnel, infrastructure, and care delivery models. A 42-item web-based survey was administered to medical directors of transplantation centers in the United States that reported any allogeneic HCT to the Center for International Blood and Marrow Transplant Research in 2011. The response rate for the survey was 79% for adult programs (85 of 108 centers) and 82% for pediatric programs (54 of 66 centers). For describing results, we categorized centers into groups with similar volumes based on 2010 total HCT activity (adult centers, 9 categories; pediatric centers, 6 categories). We observed considerable variation in available resources, infrastructure, personnel, and care delivery models among adult and pediatric transplantation centers. Characteristics varied substantially among centers with comparable transplantation volumes. Transplantation centers may find these data helpful in assessing their present capacity and use them to evaluate potential resource needs for personnel, infrastructure, and care delivery and in planning for growth.

Effect of race on outcomes after allogeneic hematopoietic cell transplantation for severe aplastic anemia

We compared outcomes after hematopoietic cell transplantation in patients of African American (n = 84) and Caucasian (n = 215) descent with severe aplastic anemia. African Americans and Caucasians were matched for age, donor–recipient human leukocyte antigen match, graft type, and transplantation year. The median follow‐up of surviving patients was 5 years. In multivariate analysis, overall mortality risks were higher for African Americans compared to Caucasians (relative risk 1.73, P = 0.01). The 5‐year probabilities of overall survival adjusted for interval from diagnosis to transplantation, and performance score was 58% for African Americans and 73% for Caucasians. The day‐100 cumulative incidence of grade III–IV, but not grade II–IV acute graft‐versus‐host disease (GVHD), was higher in African Americans compared to Caucasians (29% vs. 13%, P = 0.006). Although the 5‐year cumulative incidence of chronic GVHD was not significantly different between the racial groups, African Americans were more likely to have extensive chronic GVHD compared to Caucasians (72% vs. 49%, P = 0.06). Survival differences between Caucasians and African Americans can be attributed to multiple factors. Our data suggest that some of the observed survival differences between Caucasians and African Americans may be explained by higher rates of acute GVHD and severity of chronic GVHD. Am. J. Hematol. 89:125–129, 2014.

Hematopoietic cell transplantation in Latin America

Abstract The first bone marrow transplantation in Latin America was performed more than 30 years ago and since then several countries have started transplant programs. The Center for International Blood and Marrow Transplant Research captured information on 13 473 transplants performed in Latin America from 1981 to 2009. The current report summarizes this activity. Argentina, Mexico, and Brazil have the largest activity in the region. Despite increase in the annual number of transplants, the activity is limited to sibling donor and autologous transplants. Indications are similar to other regions with a proportionally higher number of pediatric transplants for treatment of non-malignant diseases. Unrelated donor transplant activity is also increasing through collaborations with international donor registries and the development of the first national donor registry in Brazil. Umbilical cord transplants were also reported in Latin American centers, mainly in Brazil and most commonly used for treatment of children with malignant diseases. In conclusion, hematopoietic cell transplantation is routinely performed in several centers in Latin America. However, the activity is low compared to the population in need. Challenges with costs of transplantation, donor availability, number of centers of excellence, and trained personnel need to be addressed for further development of this field in the region. Additionally, more integration between countries and transplant centers is an important next step and can assist in improving awareness for the field and maximizing the transplant activity in Latin America.

Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases.

Advances in allogeneic hematopoietic cell transplantation for sickle cell disease have improved outcomes, but there is limited analysis of healthcare utilization in this setting. We hypothesized that, compared to late transplantation, early transplantation (at age <10 years) improves outcomes and decreases healthcare utilization. We performed a retrospective study of children transplanted for sickle cell disease in the USA during 2000–2013 using two large databases. Univariate and Cox models were used to estimate associations of demographics, sickle cell disease severity, and transplant-related variables with mortality and chronic graft-versus-host disease, while Wilcoxon, Kruskal-Wallis, or linear trend tests were applied for the estimates of healthcare utilization. Among 161 patients with a 2-year overall survival rate of 90% (95% confidence interval [CI] 85–95%) mortality was significantly higher in those who underwent late transplantation versus early (hazard ratio (HR) 21, 95% CI 2.8–160.8, P=0.003) and unrelated compared to matched sibling donor transplantation (HR 5.9, 95% CI 1.7–20.2, P=0.005). Chronic graftversus host disease was significantly more frequent among those translanted late (HR 1.9, 95% CI 1.0–3.5, P=0.034) and those who received an unrelated graft (HR 2.5, 95% CI 1.2–5.4; P=0.017). Merged data for 176 patients showed that the median total adjusted transplant cost per patient was

Alternative donor hematopoietic stem cell transplantation for sickle cell disease

467,747 (range:

Practice pattern changes and improvements in hematopoietic cell transplantation for primary immunodeficiencies

344,029–

CD34-Selected, T Cell-Depleted Alternative Donor Stem Cell Transplantation For Children

799,219). Healthcare utilization was lower among recipients of matched sibling donor grafts and those with low severity disease compared to those with other types of donor and disease severity types (P<0.001 and P=0.022, respectively); no association was demonstrated with late transplantation (P=0.775). Among patients with 2-year pre- and post-transplant data (n=41), early transplantation was associated with significant reductions in admissions (P<0.001), length of stay (P<0.001), and cost (P=0.008). Early transplant outcomes need to be studied prospectively in young children without severe disease and an available matched sibling to provide conclusive evidence for the superiority of this approach. Reduced post-transplant healthcare utilization inpatient care indicates that transplantation may provide a sustained decrease in healthcare costs over time.

The Effect of Transplant Center Characteristics On Survival After Pediatric Hematopoietic Cell Transplantation

Most patients who could be cured of sickle cell disease (SCD) with stem cell transplantation do not have a matched sibling donor. Successful use of alternative donors, including mismatched family members, could provide a donor for almost all patients with SCD. The use of a reduced-intensity conditioning regimen may decrease late adverse effects. Ten patients with symptomatic SCD underwent CD34+ cell-selected, T-cell-depleted peripheral blood stem cell transplantation from a mismatched family member or unrelated donor. A reduced-intensity conditioning regimen including melphalan, thiotepa, fludarabine, and rabbit anti-thymocyte globulin was used. Patients were screened for a companion study for immune reconstitution that included a donor lymphocyte infusion given 30-42 days after transplant with intravenous methotrexate as graft-versus-host disease (GVHD) prophylaxis. Seven eligible patients were treated on the companion study. Nine of 10 patients are alive with a median follow-up of 49 months (range, 14-60 months). Surviving patients have stable donor hematopoietic engraftment (mean donor chimerism, 99.1% ± 0.7%). There were no sickle cell complications after transplant. Two patients had grade II-IV acute GVHD. One patient had chronic GVHD. Epstein-Barr virus-related posttransplant lymphoproliferative disorder (PTLD) occurred in 3 patients, and 1 patient died as a consequence of treatment of PTLD. Two-year overall survival was 90%, and event-free survival was 80%. A reduced-intensity conditioning regimen followed by CD34+ cell-selected, T-cell-depleted alternative donor peripheral blood stem cell transplantation achieved primary engraftment in all patients with a low incidence of GVHD, although PTLD was problematic. This trial was registered at clinicaltrials.gov as #NCT00968864.

Transplant Conditioning Regimens and Outcomes After Allogeneic Hematopoietic Cell Transplantation (HCT) In Children and Adolescents with Acute Lymphoblastic Leukemia (ALL)

Allogeneic HCT practice patterns for PID changed between 1974-2016. Three-year survival improved to ≥70% for SCID and non-SCID patients after 1999, and further increased to 94% for SCID patients transplanted 2010-2016 following newborn screening diagnosis.