Comparing the cost of preparing matched unrelated donor and TCR α+ ß+ /CD19+ depleted donor material for pediatric hematopoietic stem cell transplants in Australia.

Use of TCR α+ ß+ /CD19+ depletion in a pediatric setting has improved the utility of haploidentical donor material, resulting in better rates of engraftment, lower rates of graft vs host disease (GVHD), and improved transplant-related mortality. There are currently no data available on the costs of TCR α+ ß+ /CD19+ depletion. This study assessed the costs of acquiring and preparing TCR α+ ß+ /CD19+ depleted haploidentical donor cells in comparison with matched unrelated donor (MUD) products for use in pediatric patients in Australia. Data from four pediatric transplant centers were used to estimate the resources required for donor work-up, graft acquisition, and laboratory procedures for graft preparation. Information on MUD work-up and graft acquisition was also acquired from these sites and from the national coordinating donor center in Australia. Australian-specific prices and fees were used to estimate total average costs for each transplant type, converted to USD. Preparation of graft material (including work-up, acquisition, and laboratory processes) costs USD 28 963 for TCR α+ ß+ /CD19+ depleted haploidentical grafts and USD 27 297 for MUD grafts. The estimated difference of USD 1666 is largely attributed to the process and consumables to perform TCR α+ ß+ /CD19+ depletion. Given the potential for recipients of TCR α+ ß+ /CD19+ depleted grafts to require minimal GVHD prophylaxis and experience less transplant-related morbidity and mortality, use of TCR α+ ß+ /CD19+ depletion appears favorable despite the higher initial cost. Research is currently ongoing to assess the clinical effectiveness and potential cost-effectiveness of TCR α+ ß+ /CD19+ depletion over a patients' lifetime.

Hematopoietic stem cell transplantation in the Sultanate of Oman.

The Sultanate of Oman is one of the Arabian Gulf countries with a total population of 4,414,051 as of mid 2016, of which 2,427,825 are Omanis. The gross national income per capita was 7327.7 RO (Omani rial; equivalent to US$19,033) in 2014. There are two hematopoietic stem cell transplantation (HSCT) centers in Oman: the Sultan Qaboos University Hospital (SQUH; allogeneic and autologous) and the Royal Hospital (RH; autologous). HSCT activity in Oman started in 1995 at the SQUH center, which had only one bed, and four cases were performed in that year. The number of allogeneic HSCTs at the SQUH ranged between four and 29 cases per year, of which malignancy was the main indication for transplantation (47%). Most of the transplants were performed from identical sibling donor. T-deplete haploidentical and recently T-replete haploidentical HSCT were also performed at the SQUH center. In the allogeneic HSCT cohort transplanted at the SQUH, the risk of acute graft-versus-host disease (Grades II-IV) was 18%, whereas the risk of extensive chronic graft-versus-host disease was 8%. The HSCT unit at the RH, which started in 2014, performs autologous HSCT procedures only. The number of autologous HSCT cases at the RH ranged between three and 16 cases per year. Limited bed availability is a frequent obstacle to HSCT in Oman. Construction of a much larger national HSCT center is about to be completed, which will likely improve access to transplant services in Oman.

Health-related quality of life in severe cryoglobulinaemic vasculitis and improvement after B-cell depleting therapy.

OBJECTIVES: To study the health-related quality of life (HRQOL) in severe cryoglobulinaemic vasculitis (CV) associated with hepatitis C virus infection (HCV) and to describe the effect of rituximab on HRQOL. METHODS: HRQOL was evaluated with the Medical Outcomes Study Short Form 36 (SF-36). Health Survey questionnaire was submitted to 15 patients with severe CV. SF-36 questionnaire was evaluated at baseline and after rituximab. Physical Health Composite Summary (PCS) and Mental Health Composite Summary (MCS) scores were calculated according to standard protocols, and normalised to healthy controls. SF-36 summary scores were compared with those of HCV positive patients without CV, and other vasculitis published in the literature. European Quality of Life-5 dimensions (EQ5D) scores were also derived. RESULTS: Physical and mental domain scores were all reduced if compared with those of the healthy population, with physical domains being greatly affected. HRQOL of CV was comparable with HRQOL reported for the other small vessel vasculitis. The development of CV in HCV positive patients worsened PCS rather than MCS score. Birmingham Vasculitis Activity Score (BVAS) did not correlate with HRQOL, while the presence of peripheral neuropathy was associated with a worse HRQOL. Early rituximab treatment improved both PCS and MCS scores, with long-term effects. CONCLUSIONS: PCS rather than MCS was affected in HCV positive patients when CV is present. Rituximab improved both physical and mental domains, thus supporting its use before antiviral therapy in severe HCV-related CV. The cost/benefits ratio of a sequential therapy may be supported.

Economic analysis of unrelated allogeneic bone marrow transplantation: results from the randomized clinical trial of T-cell depletion vs unmanipulated grafts for the prevention of graft-versus-host disease.

Unrelated-donor marrow transplantation is a potential option for transplant candidates lacking a compatible related donor. The T-cell Depletion Study compared the 3-year disease-free survival for patients receiving T-cell-depleted (TCD) donor marrow (n = 203) vs unmanipulated donor marrow with methotrexate and cyclosporine (M/C) (n = 207). Hospital costs during index admission were documented with billing data, while hospital costs during subsequent 6-month follow-up were estimated from case report forms. Patients with index admission billing were included in the analysis (TCD = 119, M/C = 127). Total hospital length of stay (LOS) was similar across groups, with medians 47.0 days for TCD and 52.0 days for M/C (P = 0.72). Total hospital costs were comparable, 145,115 dollars vs 141,981 dollars (P = 0.63) for TCD and M/C, respectively. However, controlling for site and patient characteristics, TCD was associated with a 12.1% reduction in LOS for the index admission (95% CI -19.4%, -4.3%). Independent of treatment, HLA matching (6/6) was associated with an 8.6% (95% CI -17.4%, +1.2%) reduction in the index admission LOS, while cost was lower by 15.8% (95% CI -26.7%, -3.3%). Treatment costs were similar for TCD and M/C study groups. Savings on reduced cost for treating acute graft-versus-host disease were likely offset by increase in serious infections in the TCD arm.

Comparison of T-cell-depleted and non-T-cell-depleted unrelated donor transplantation for hematologic diseases: clinical outcomes, quality of life, and costs.

T-cell depletion (TCD) and immunosuppressive medications (ISTs) are 2 methods used for graft-versus-host disease (GVHD) prophylaxis in unrelated donor (URD) transplantation. However, comparisons of the clinical outcomes including quality of life and direct medical costs associated with each type of procedure have not been reported. We reviewed 48 TCD and 98 IST procedures performed from 1/1/97 to 12/31/99 at the Dana-Farber Cancer Institute, Boston, MA. With a median follow-up of 1.5 years for survivors, no differences were seen in relapse, acute GVHD, and overall survival between TCD and IST patients. Multivariable Cox modeling showed that age of 50 years or less (P =.002) and low-risk disease (P =.001) predicted survival, but method of GVHD prophylaxis (P =.6) and degree of human leukocyte antigen (HLA) matching (P =.8) did not. A subset of patients (53%) completed quality of life surveys prior to and at 6 and 12 months after transplantation; participation in the quality of life study was not associated with clinical characteristics and outcomes. No differences were seen in quality of life scores prior to transplantation, and changes over time were similar between groups. Costs ($113 000 vs $155 000, P <.0001) and total hospital days (34 vs 46, P =.0006) were significantly lower for patients undergoing TCD procedures. As prospective, randomized studies comparing methods of GVHD prophylaxis are performed, assessment of quality of life and costs should be included to fully understand the overall impact of each intervention.

Leukocyte depletion of cellular blood components.

Clinical studies have indicated that the use of leukocyte-reduced cellular blood components produced in the laboratory may prevent febrile reactions and delay or prevent alloimmunization to HLA antigens and refractoriness to platelet transfusion. Additional investigations regarding the effects of the use of leukocyte-reduced blood components were reported during the past year. A recent study in patients with hematologic malignancy that employed the commonly used bedside leukocyte-reduction filters failed to confirm a decrease in the rate of alloimmunization, except in a subgroup of patients with acute myelogenous leukemia. Another major multicenter trial confirmed the effectiveness of leukocyte-reduced blood components in the prevention of cytomegalovirus infection. The effect of allogeneic leukocytes in transfused blood on immune function in patients undergoing colorectal surgery continues to receive attention. Whereas one study failed to demonstrate an adverse effect of standard blood components on disease recurrence or survival, a second study demonstrated a marginally significant decrease in infectious complications in patients who received only leukocyte-reduced blood. Increasingly efficient leukocyte-reduction filters have been developed for cellular blood components, many of which are best suited for laboratory filtration of unstored blood. Laboratory studies indicate that prestorage leukocyte-reduction of cellular blood components does not impair erythrocyte or platelet function and will not increase the incidence of microbial contamination of blood. New methods that employ flow cytometry should enable improved quality control of blood components rendered leukocyte-reduced by the newer, more efficient filters. Finally, a cost-benefit analysis suggests that the appropriate use of leukocyte-reduction filters for acute leukemia patients may reduce the cost of health care to these patients.