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1.
Biol Blood Marrow Transplant ; 25(2): 233-238, 2019 02.
Article in English | MEDLINE | ID: mdl-30219699

ABSTRACT

Filgrastim (FIL) is the most common growth factor combined with plerixafor for autologous hematopoietic progenitor cell mobilization, but requires daily, multi-injection administration. We adopted a standardized mobilization regimen with pegfilgrastim (PEG) and upfront plerixafor, allowing for a single injection given the long half-life and slow elimination of PEG. Between 2015 and 2017, a total of 235 patients with lymphoma or plasma cell dyscrasias underwent mobilization with PEG 6 mg on day 1 and upfront plerixafor 24 mg on day 3, followed by apheresis on day 4 regardless of peripheral blood CD34+ cells. The median CD34+ cells/mm3 in peripheral blood on first day of collection was 48 and median collection yield was 7.27 × 106 CD34+ cells/kg (range, 0.32 to 39.6 × 106 CD34+ cells/kg) after a mean of 1.6 apheresis collections. Overall, 83% of patients achieved the mobilization target, and 95% reached the minimum necessary CD34+ cell yield to proceed with transplantation (2 × 106 CD34+ cells/kg). Because FIL is weight-based and dosed daily, the cost comparison with PEG is influenced by patient weight and number of apheresis sessions required. A cost simulation using actual patient data indicates that PEG is associated with lower cost than FIL for the majority of patients. Autologous hematopoietic progenitor cell mobilization with PEG and plerixafor is practical, effective, and not associated with increased cost compared with FIL mobilization.


Subject(s)
Costs and Cost Analysis , Filgrastim , Hematopoietic Stem Cell Mobilization/economics , Lymphoma , Peripheral Blood Stem Cell Transplantation/economics , Polyethylene Glycols , Adult , Aged , Female , Filgrastim/administration & dosage , Filgrastim/economics , Humans , Lymphoma/economics , Lymphoma/pathology , Lymphoma/therapy , Male , Middle Aged , Polyethylene Glycols/administration & dosage , Polyethylene Glycols/economics , Transplantation, Autologous
3.
Transplant Proc ; 49(1): 4-9, 2017.
Article in English | MEDLINE | ID: mdl-28104154

ABSTRACT

BACKGROUND: When assessing the cost of transplants in Japan, earlier studies have been limited to case series that investigated inpatient cost alone. Few studies have evaluated total cost, which includes inpatient, outpatient, and pharmaceutical costs, or compared costs before and after transplantation. Using the National Database of Health Insurance Claims and Specific Health Checkups of Japan (NDB), we investigated the total cost of major transplantation and contributing factors. METHODS: We analyzed the cost and complications of patients who underwent a cadaveric renal transplantation (CRT), living renal transplantation (LRT), living-donor liver transplantation (LDLT), allogeneic bone marrow transplantation, autologous bone marrow transplantation, allogeneic peripheral blood stem cell transplantation, or autologous peripheral blood stem cell transplantation (auto-PBSCT) from April 2009 to March 2010. RESULTS: The highest total cost of the month of transplantation was 4.95 million yen (JPY) for LDLT. Among renal transplantations, the cost of CRT was higher than LRT (3.69 vs 3.55 million JPY). Recipients of auto-PBSCT complicated by graft-versus-host disease, urinary tract infection, sepsis, or pneumonia had a significantly higher average total cost during the month of transplantation and the 2 following months than patients without it, as well as statistically longer total treatment days. CONCLUSIONS: In Japan, almost all medical services are covered by national health insurance, and the Japan government has begun to allow the use of the NDB for research activities. This is the first study to use the NDB to analyze the cost of transplantation, with technical and institutional limitations.


Subject(s)
Bone Marrow Transplantation/economics , Kidney Transplantation/economics , Liver Transplantation/economics , Peripheral Blood Stem Cell Transplantation/economics , Postoperative Complications/economics , Bone Marrow Transplantation/methods , Costs and Cost Analysis , Databases, Factual , Female , Humans , Insurance, Health/statistics & numerical data , Japan , Kidney Transplantation/methods , Liver Transplantation/methods , Male , Peripheral Blood Stem Cell Transplantation/adverse effects , Peripheral Blood Stem Cell Transplantation/methods , Postoperative Complications/etiology
4.
Bone Marrow Transplant ; 50(5): 663-7, 2015 May.
Article in English | MEDLINE | ID: mdl-25730190

ABSTRACT

Outcomes in multiple myeloma (MM) have improved significantly with novel agent therapy and autologous stem cell transplantation (ASCT). ASCTs are typically planned as either tandem or a single transplant with additional stored PBSCs available for a second salvage transplant. To accommodate these strategies, many centers routinely collect and store adequate PBSCs for two ASCTs. We analyzed the cost associated with this practice by determining the expenses of PBSC collection, cryopreservation and storage, and the ultimate use of additional cryopreserved PBSCs in patients who had undergone at least one ASCT. There were 889 MM patients transplanted between 1993 and 2011 at our center. Most (N=726) had residual PBSCs in storage after their first ASCT (ASCT1). Only 135 patients underwent a second ASCT within a median of 14 months after ASCT1. The percentage of patients receiving a second ASCT declined over time. The resources required to collect and store unused PBSCs added up to 336 extra patient days of apheresis and 41 587 extra patient months of cryopreservation, translating into an average extra cost per patient of US$4981.12. A reconsideration of conventional PBSC collection and storage practices would save significant cost for the majority of MM patients who never undergo a second ASCT.


Subject(s)
Blood Component Removal , Cryopreservation , Multiple Myeloma , Peripheral Blood Stem Cell Transplantation , Adult , Aged , Autografts , Blood Component Removal/economics , Blood Component Removal/methods , Costs and Cost Analysis , Cryopreservation/economics , Cryopreservation/methods , Female , Follow-Up Studies , Humans , Male , Middle Aged , Multiple Myeloma/economics , Multiple Myeloma/therapy , Peripheral Blood Stem Cell Transplantation/economics , Peripheral Blood Stem Cell Transplantation/methods , Retrospective Studies
5.
Br J Haematol ; 164(1): 113-23, 2014 Jan.
Article in English | MEDLINE | ID: mdl-24138497

ABSTRACT

To date, no prospective study on Plerixafor 'on-demand' in combination with chemotherapy and granulocyte colony-stimulating factor (G-CSF) has been reported. We present an interim analysis of the first prospective study in which Plerixafor was administered on-demand in patients affected by multiple myeloma and lymphoma who received high dose cyclophosphamide or DHAP (dexamethasone, cytarabine, cisplatin) plus G-CSF to mobilize peripheral blood stem cells (PBSC). One hundred and two patients were evaluable for response. A cohort of 240 patients receiving the same mobilizing chemotherapy was retrospectively studied. Failure to mobilize CD34(+) cells in peripheral blood was reduced by 'on-demand' strategy compared to conventional mobilization; from 13·0 to 3·0% (P = 0·004). Failure to harvest CD34(+) cells 2 × 10(6) /kg decreased from 20·9 to 4·0% (P = 0·0001). The on-demand Plerixafor strategy also resulted in a lower rate of mobilization failure (P = 0·03) and harvest failure (P = 0·0008) when compared to a 'bias-adjusted set of controls'. Evaluation of economic costs of the two strategies showed that the overall cost of the two treatments were comparable when salvage mobilizations were taken into account. When in combination with cyclophosphamide or DHAP plus G-CSF, the 'on-demand' use of Plerixafor showed, in comparison to conventionally treated patients, a significant improvement in mobilization of PBSC with no increase in overall cost.


Subject(s)
Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Granulocyte Colony-Stimulating Factor/administration & dosage , Hematopoietic Stem Cell Mobilization/methods , Heterocyclic Compounds/administration & dosage , Lymphoma/therapy , Multiple Myeloma/therapy , Peripheral Blood Stem Cell Transplantation/methods , Adult , Aged , Benzylamines , Blood Component Removal/economics , Blood Component Removal/methods , Cyclams , Female , Granulocyte Colony-Stimulating Factor/economics , Hematopoietic Stem Cell Mobilization/economics , Heterocyclic Compounds/economics , Humans , Lymphoma/drug therapy , Lymphoma/surgery , Male , Middle Aged , Multiple Myeloma/drug therapy , Multiple Myeloma/surgery , Peripheral Blood Stem Cell Transplantation/economics , Prospective Studies , Retrospective Studies , Young Adult
6.
Stem Cells Dev ; 22 Suppl 1: 103-10, 2013 Dec.
Article in English | MEDLINE | ID: mdl-24304086

ABSTRACT

Cord blood (CB) has been used since 1988 as a source of hematopoietic stem cells (HSCs) and progenitor cells for hematopoietic cell transplantation (HCT) to treat patients with malignant and nonmalignant disorders. CB has both advantages and disadvantages when compared with other tissue sources of HSCs such as bone marrow and mobilized peripheral blood, which are also being used in the setting of HCT. This short review focuses on some historical information, as well as current efforts that are being assessed to enhance the efficacy of CB HCT. Also of importance are the costs of CB, and the feasibility and economics of using such to be identified, and newly confirmed improvements worldwide for the greatest number of patients. In this context, simple methods that would not necessarily entail the need for selected cell-processing facilities to ex vivo expand or improve the CB graft's functional activity may be of interest, with one such possibility being the use of an orally active inhibitor of the enzyme dipeptidylpeptidase 4, alone or in combination with other new and innovative approaches for improving HSC engraftment and in vivo repopulating capability of CB.


Subject(s)
Cord Blood Stem Cell Transplantation/economics , Hematopoietic Stem Cell Transplantation/economics , Peripheral Blood Stem Cell Transplantation/economics , Fetal Blood/cytology , Hematopoietic Stem Cells/cytology , Humans
7.
PLoS One ; 8(9): e75047, 2013.
Article in English | MEDLINE | ID: mdl-24098678

ABSTRACT

BACKGROUND: High-dose therapy with autologous peripheral stem cell transplantation represents today the standard approach for younger multiple myeloma patients. This study aimed to evaluate the long term economic impact of autologous transplantation with respect to conventional therapy. METHODS: We retrospectively reviewed the charts of multiple myeloma patients diagnosed at our department between 1986 and 2003 and treated according to the therapy considered standard at the time of diagnosis. Analysis of costs was done by assessing resource utilization and direct costs were measured and monetized before proceeding with the analysis, based on public health service tariffs. RESULTS: Group A including 78 patients treated with Melphalan and Prednisone was compared with Group B including 74 patients who received an autologous transplant. The median overall survival was 3.2 and 5.4 years respectively (p = 0.0002). Mean cost per patient was significantly higher in group B with respect to group A (102373€ vs 23825€; p<0.001). The final quality-adjusted-life-year gain in group B patients as compared to group A was 1.73 QALY, with an incremental cost-effectiveness ratio of 45460€. With a threshold of 75000€ per QALY gained, the cost effectiveness acceptability curve indicated that the probability that autologous transplantation in multiple myeloma is a cost-effective intervention is 90%. CONCLUSIONS: The cost of autologous transplantation remains high. The calculated incremental cost-effectiveness ratio, however, given the significant prolongation of overall survival obtained with autologous transplantation, is within an acceptable threshold. Notwithstanding, its high cost should be taken into account when considering the whole cost of multiple myeloma.


Subject(s)
Multiple Myeloma/therapy , Peripheral Blood Stem Cell Transplantation/economics , Peripheral Blood Stem Cell Transplantation/methods , Adult , Cost-Benefit Analysis , Female , Humans , Italy , Male , Melphalan/therapeutic use , Middle Aged , Multiple Myeloma/drug therapy , Prednisone/therapeutic use , Retrospective Studies , Statistics, Nonparametric , Survival Analysis
8.
Transfus Apher Sci ; 49(3): 615-22, 2013 Dec.
Article in English | MEDLINE | ID: mdl-23973731

ABSTRACT

Scarce information is available about the cost of mobilisation/collection of peripheral blood stem cells for patients undergoing autologous transplant for relapsed Lymphoma or Multiple Myeloma. This paper reports the consumption of resources and costs collected through a survey among Italian Centres who adhere to the GITMO and SIdEM scientific societies. General transplant information was extracted from the European Promise database. Resources used alongside the phases of mobilisation/collection were retrieved. Resources for each of the process phases were quantified and averaged across centres and a unit cost value was attributed, based on administrative data from 3 centres, tariffs and market values. 25/89 Centres (34% of 2009 Promise transplants) provided data according to their standard practice. The mean cost/patient of the process of cell mobilisation/collection was € 6830 ± 1802 for Multiple Myeloma and € 7304 ± 1542 for Lymphoma. The organisational path for PBSC mobilisation/collection appears complex and cumbersome, spread amongst different treatment settings, with many different healthcare professionals being involved and considerable amounts of time and resources being currently dedicated to the management of patients requiring autologous transplantation.


Subject(s)
Hematopoietic Stem Cell Mobilization/economics , Hematopoietic Stem Cell Transplantation/economics , Lymphoma/surgery , Multiple Myeloma/surgery , Peripheral Blood Stem Cell Transplantation/economics , Data Collection , European Union , Female , Health Care Costs , Hematopoietic Stem Cell Mobilization/methods , Hematopoietic Stem Cell Transplantation/methods , Humans , Italy , Lymphoma/economics , Male , Middle Aged , Multiple Myeloma/economics , Peripheral Blood Stem Cell Transplantation/methods , Surveys and Questionnaires , Transplantation Conditioning , Transplantation, Autologous
9.
J Clin Apher ; 28(6): 395-403, 2013 Dec.
Article in English | MEDLINE | ID: mdl-23922227

ABSTRACT

Peripheral blood stem cells (PBSCs) are preferred source of hematopoietic stem cells for autologous transplantation. Mobilization of PBSCs using chemotherapy and/or granulocyte colony-stimulating factor (G-CSF) however fails in around 20%. Combining G-CSF with plerixafor increases the mobilizations success. We compared cost-effectiveness of following schemes: the use of plerixafor "on demand" (POD) during the first mobilization in all patients with inadequate response, the remobilization with plerixafor following failure of the first standard mobilization (SSP), and the standard (re)mobilization scheme without plerixafor (SSNP). Decision tree models populated with data from a first-of-a-kind patient registry in six Czech centers (n = 93) were built to compare clinical benefits and direct costs from the payer's perspective. The success rates and costs for POD, SSP and SSNP mobilizations were; 94.9%, $7,197; 94.7%, $8,049; 84.7%, $5,991, respectively. The direct cost per successfully treated patient was $7,586, $8,501, and $7,077, respectively. The cost of re-mobilization of a poor mobilizer was $5,808 with G-CSF only and $16,755 if plerixafor was added. The total cost of plerixafor "on-demand" in the sub-cohort of poor mobilizers was $17,120. Generally, plerixafor improves the mobilization success by 10% and allows an additional patient to be successfully mobilized for incremental $11,803. Plerixafor is better and cheaper if used "on demand" than within a subsequent remobilization.


Subject(s)
Granulocyte Colony-Stimulating Factor/economics , Hematopoietic Stem Cell Mobilization/economics , Heterocyclic Compounds/economics , Lymphoma/economics , Multiple Myeloma/economics , Peripheral Blood Stem Cell Transplantation/economics , Adolescent , Adult , Aged , Benzylamines , Child , Child, Preschool , Cost-Benefit Analysis , Cyclams , Cytapheresis/statistics & numerical data , Czechoslovakia , Decision Trees , Female , Granulocyte Colony-Stimulating Factor/therapeutic use , Health Expenditures , Hematopoietic Stem Cell Mobilization/methods , Heterocyclic Compounds/therapeutic use , Humans , Infant , Length of Stay/statistics & numerical data , Lymphoma/surgery , Male , Middle Aged , Models, Economic , Multiple Myeloma/surgery , Treatment Outcome , Young Adult
10.
Biol Blood Marrow Transplant ; 19(1): 87-93, 2013 Jan.
Article in English | MEDLINE | ID: mdl-22922211

ABSTRACT

Historically, up to 30% of patients were unable to collect adequate numbers of peripheral blood stem cells (PBSCs) for autologous stem cell transplantation (ASCT). Plerixafor in combination with granulocyte colony-stimulating factor (G-CSF) has shown superior results in mobilizing peripheral blood (PB) CD34+ cells in comparison to G-CSF alone, but its high cost limits general use. We developed and evaluated risk-adapted algorithms for optimal utilization of plerixafor. In plerixafor-1, PBSC mobilization was commenced with G-CSF alone, and if PB CD34 on day 4 or day 5 was <10/µL, plerixafor was administered in the evening, and apheresis commenced the next day. In addition, if on any day, the daily yield was <0.5 × 10(6) CD34/kg, plerixafor was added. Subsequently, the algorithm was revised (plerixafor-2) with lower thresholds. If day-4 PB CD34 <10/µL for single or <20/µL for multiple transplantations, or day-1 yield was <1.5 × 10(6) CD34/kg, or any subsequent daily yield was <0.5 × 10(6) CD34/kg, plerixafor was added. Three time periods were analyzed for results and associated costs: January to December 2008 (baseline cohort; 319 mobilization attempts in 278 patients); February to November 2009 (plerixafor-1; 221 mobilization attempts in 216 patients); and December 2009 to June 2010 (plerixafor-2; 100 mobilization attempts in 98 patients). Plerixafor-2 shows a significant improvement in PB CD34 collection, increased number of patients reaching minimum and optimal goals, fewer days of apheresis, and fewer days of mobilization/collection, albeit at increased costs. In conclusion, although the earlier identification of ineffective PBSC mobilization and initiation of plerixafor (plerixafor-2) increases the per-patient costs of PBSC mobilization, failure rates, days of apheresis, and total days of mobilization/collection are lower.


Subject(s)
Algorithms , Hematopoietic Stem Cell Mobilization/economics , Heterocyclic Compounds/economics , Peripheral Blood Stem Cell Transplantation/economics , Adult , Aged , Benzylamines , Case-Control Studies , Costs and Cost Analysis , Cyclams , Female , Hematopoietic Stem Cell Mobilization/methods , Heterocyclic Compounds/administration & dosage , Heterocyclic Compounds/adverse effects , Humans , Lymphoma, Non-Hodgkin/economics , Lymphoma, Non-Hodgkin/therapy , Male , Middle Aged , Plasmacytoma/economics , Plasmacytoma/therapy , Risk Factors , Time Factors , Transplantation, Autologous
11.
Eur J Cancer ; 48(5): 713-20, 2012 Mar.
Article in English | MEDLINE | ID: mdl-22248711

ABSTRACT

AIM: To evaluate in a multicentre randomised study the effect on duration of febrile neutropenia (FN), the safety and cost-effectiveness of a single subcutaneous pegfilgrastim injection compared with daily injections of filgrastim after peripheral blood stem cell transplantation in patients receiving high dose chemotherapy for myeloma and lymphoma. METHODS: Patients were randomly assigned to a single dose of pegfilgrastim at day 5 (D5) or daily filgrastim from D5 to the recovery of absolute neutrophil count (ANC) to 0.5 G/L. Duration of FN, of neutrophil and platelet recovery, transfusion and antibiotic requirements were the main end-points of the study. Costs were calculated from D0 until transplant unit discharge. The incremental cost-effectiveness ratio was expressed as the cost per day of FN prevented. Probabilistic sensitivity analysis was performed by non-parametric bootstrap methods. RESULTS: Between October 2008 and September 2009, 10 centres enrolled 151 patients: 80 patients with lymphoma and 71 patients with myeloma. The mean duration of FN was 3.07 days (standard deviation (SD) 1.96) in the pegfilgrastin arm and 3.29 (SD 2.54) in the filgrastim one. Mean total costs were 23,256 and 25,448 euros for pegfilgrastim and filgrastim patients, respectively. There was a 62% probability that pegfilgrastim strictly dominates filgrastim. CONCLUDING STATEMENT: Pegfilgrastim after PBSC transplantation in myeloma and lymphoma is safe, effective when compared with filgrastim and could represent a cost-effective alternative in this setting.


Subject(s)
Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Granulocyte Colony-Stimulating Factor/therapeutic use , Lymphoma/therapy , Multiple Myeloma/therapy , Neutropenia/drug therapy , Peripheral Blood Stem Cell Transplantation/methods , Adult , Aged , Antineoplastic Combined Chemotherapy Protocols/economics , Combined Modality Therapy , Cost-Benefit Analysis , Female , Fever/drug therapy , Fever/economics , Fever/etiology , Filgrastim , Granulocyte Colony-Stimulating Factor/economics , Humans , Lymphoma/drug therapy , Lymphoma/surgery , Male , Middle Aged , Multiple Myeloma/drug therapy , Multiple Myeloma/surgery , Neutropenia/economics , Neutropenia/etiology , Peripheral Blood Stem Cell Transplantation/adverse effects , Peripheral Blood Stem Cell Transplantation/economics , Polyethylene Glycols , Recombinant Proteins/economics , Recombinant Proteins/therapeutic use , Young Adult
13.
Transfusion ; 51(10): 2175-82, 2011 Oct.
Article in English | MEDLINE | ID: mdl-21492180

ABSTRACT

BACKGROUND: Plerixafor is a recently Food and Drug Administration (FDA)-approved CXCR4 antagonist, which is combined with granulocyte-colony-stimulating factor (G-CSF) to facilitate stem cell mobilization of lymphoma and myeloma patients. STUDY DESIGN AND METHODS: To evaluate the effectiveness and the related costs of a "just-in-time" strategy of plerixafor administration, we performed a retrospective cohort study comparing 148 consecutive lymphoma and myeloma patients in whom mobilization was attempted during 2008 before the Food and Drug Administration (FDA) approval of plerixafor with 188 consecutive patients mobilized during 2009 after FDA approval. RESULTS: Plerixafor was administered to 64 of 188 patients considered to be at risk for mobilization failure due to either their medical history ("high risk," n = 23) or the occurrence of peripheral blood CD34+ count of fewer than 15 × 10(6) cells/L with a white blood cell count of greater than 10 × 10(9) cells/L after at least 5 days of G-CSF administration (just-in-time, n = 41). The success rates of collecting a minimum transplant CD34+ cell dose (≥2 × 10(6) cells/kg) or target cell dose (≥5 × 10(6) lymphoma or ≥10 × 10(6) CD34+ cells/kg myeloma) in the just-in-time patients compared favorably with the 36 poor mobilizers collected with G-CSF alone: 93% versus 72% and 42% versus 22%, respectively. CONCLUSIONS: The use of plerixafor in selected high-risk patients and poor mobilizers did not increase the total charges associated with stem cell collection when compared with poor mobilizers treated with G-CSF alone. The targeted use of plerixafor increased the overall success rate of mobilizing a minimum number of CD34+ cells from 93% to 98% in patients with hematologic malignancies scheduled for autotransplant and increased the overall charges associated with stem cell collection in all patients by an average of 17%.


Subject(s)
Hematopoietic Stem Cell Mobilization/methods , Heterocyclic Compounds/administration & dosage , Lymphoma/therapy , Multiple Myeloma/therapy , Peripheral Blood Stem Cell Transplantation/methods , Receptors, CXCR4/antagonists & inhibitors , Salvage Therapy , Adolescent , Adult , Aged , Antigens, CD34/blood , Benzylamines , Blood Component Removal , Cohort Studies , Cost-Benefit Analysis , Cyclams , Drug Administration Schedule , Female , Granulocyte Colony-Stimulating Factor/administration & dosage , Hematopoietic Stem Cell Mobilization/economics , Hematopoietic Stem Cells/drug effects , Heterocyclic Compounds/pharmacology , Humans , Lymphoma/blood , Male , Middle Aged , Multiple Myeloma/blood , Peripheral Blood Stem Cell Transplantation/economics , Retrospective Studies , Time Factors , Transplantation, Autologous , Young Adult
14.
Biol Blood Marrow Transplant ; 16(9): 1272-81, 2010 Sep.
Article in English | MEDLINE | ID: mdl-20348004

ABSTRACT

In a retrospective study, we evaluated the cost and cost-effectiveness of allogeneic peripheral blood stem cell transplantation (PBSCT) (n = 30) compared with bone marrow transplantation (BMT) (n = 110) in children with acute leukemia after 1 year of follow-up. Treatment success was defined as disease-free survival at 1 year posttransplantation. For patients at standard risk for disease, the treatment success rate was 57.1% for PBSCT recipients and 80.3% for BMT recipients (P = not significant [NS]). The average total cost per treatment success at 1 year in the standard-risk disease group was $512,294 for PBSCT recipients and $352,885 for BMT recipients (P = NS). For patients with high-risk disease, the treatment success rate was 18.8% for PBSCT recipients and 23.5% for BMT recipients (P = NS). The cumulative average cost was $457,078 in BMT recipients and $377,316 in PBSCT recipients (P = NS). Point estimates of the incremental cost-effectiveness ratio (ICER) indicate that in patients with standard-risk disease, allogeneic BMT had lower costs and greater effectiveness than PBSCT (ICER, -$687,108; 95% confidence interval [CI], $2.4 million to dominated). For patients with high-risk disease, BMT was more effective and more costly, and it had an ICER of $1.69 million (95% CI, $29.7 million to dominated) per additional treatment success. The comparative economic evaluation provides support for BMT in standard-risk patients, but much uncertainty precludes a clear advantage of either treatment option in patients with high-risk disease. More studies using larger and randomized controlled trials are needed to confirm the long-term cost-effectiveness of each procedure.


Subject(s)
Bone Marrow Transplantation/economics , Leukemia/economics , Leukemia/therapy , Peripheral Blood Stem Cell Transplantation/economics , Acute Disease , Adolescent , Child , Child, Preschool , Cost-Benefit Analysis , Female , Humans , Infant , Infant, Newborn , Male , Retrospective Studies , Treatment Outcome
15.
Biol Blood Marrow Transplant ; 16(6): 848-53, 2010 Jun.
Article in English | MEDLINE | ID: mdl-20138227

ABSTRACT

Although the drug imatinib has been accepted as the treatment of choice for chronic myelogenous leukemia (CML) in chronic phase (CP) throughout the Western world, allogeneic stem cell transplantation (allo-SCT) continues to remain a widely practiced first-line treatment in countries with limited health care budgets. The rationale is not scientific, but largely economically based. We analyzed a cohort of 46 CML patients who participated in a graft-versus-host disease (GVHD) prophylaxis clinical trial and underwent related HLA-matched allogeneic peripheral blood stem cell transplantation. The median time of follow-up in surviving patients was 43 months (range: 4-89 months). Risk stratification of the population was done by European Blood and Marrow Transplant (EBMT) criteria. The estimated probabilities of overall survival (OS) and leukemia-free survival (LFS) at 3 years in low EBMT risk score (0-2) patients were both 91%, respectively. We conclude that in countries with restricted access to imatinib, allo-SCT should be considered early on as front-line therapy. Continued research support for bone marrow transplantation will be needed to make a global impact on this disease.


Subject(s)
Bone Marrow Transplantation/economics , Drug Therapy/economics , Leukemia, Myelogenous, Chronic, BCR-ABL Positive/drug therapy , Leukemia, Myelogenous, Chronic, BCR-ABL Positive/surgery , Adolescent , Adult , Antineoplastic Agents/economics , Antineoplastic Agents/therapeutic use , Benzamides , Bone Marrow Transplantation/methods , Child , Disease Progression , Disease-Free Survival , Female , Follow-Up Studies , Graft Survival , Graft vs Host Disease/diagnosis , Graft vs Host Disease/prevention & control , Humans , Imatinib Mesylate , Leukemia, Myelogenous, Chronic, BCR-ABL Positive/diagnosis , Male , Middle Aged , Peripheral Blood Stem Cell Transplantation/economics , Peripheral Blood Stem Cell Transplantation/methods , Piperazines/economics , Piperazines/therapeutic use , Pyrimidines/economics , Pyrimidines/therapeutic use , Recurrence , Survival Analysis , Transplantation, Homologous , Treatment Outcome , Young Adult
16.
Biol Blood Marrow Transplant ; 16(5): 678-85, 2010 May.
Article in English | MEDLINE | ID: mdl-20045479

ABSTRACT

Nonrandomized trials suggest that pegfilgrastim, a pegylated granulocyte colony-stimulating factor, could be used in lieu of filgrastim after autologus peripheral blood stem cell transplantation. This phase III, randomized, double-blinded, placebo-controlled trial compared the efficacy, costs, and safety of single-dose pegfilgrastim (single 6 mg dose) versus daily filgrastim (5 microg/kg/day) for this indication. Seventy-eight patients, matched for age, sex, underlying disease, stage, and CD34/kg transplant dose were enrolled. Cytokines were started on day +1 posttransplant and continued to an absolute neutrophil count (ANC) of 5x10(9)/L for 3 days or 10x10(9)/L for 1 day. The median time to neutrophil engraftment (ANC >1.5x10(9)/L for 3 days or 5x10(9)/L for 1 day) was the same in both groups (12 days). No differences in platelet engraftment (11 versus 13 days), number of platelet transfusions (5 versus 4), percent with positive cultures for bacterial pathogens (23% versus 15%), days of fever (1 versus 2), deaths prior to engraftment (1 versus 1), or duration of hospital stay (19 versus 19 days) were seen between the pegfilgrastim and filgrastim groups, respectively. Using the average wholesale price for doses used in this trial, there was a per-patient savings of $961 for the pegfilgrastim group (P < .001). This phase III study failed to demonstrate a difference in time to neutrophil engraftment or any clinical sequelae between pegfilgrastim and filgrastim when given post-APBSCT, with pegfilgrastim achieving a cost savings over filgrastim.


Subject(s)
Granulocyte Colony-Stimulating Factor/administration & dosage , Peripheral Blood Stem Cell Transplantation/methods , Adult , Aged , Aged, 80 and over , Blood Platelets/physiology , Costs and Cost Analysis , Double-Blind Method , Female , Filgrastim , Granulocyte Colony-Stimulating Factor/economics , Humans , Male , Middle Aged , Neutrophils/physiology , Peripheral Blood Stem Cell Transplantation/economics , Polyethylene Glycols , Recombinant Proteins , Regeneration , Transplantation, Autologous , Treatment Outcome , Young Adult
17.
Transfusion ; 50(3): 656-62, 2010 Mar.
Article in English | MEDLINE | ID: mdl-20042030

ABSTRACT

BACKGROUND: After mobilization, the collection of peripheral blood progenitor cells (PBPCs) can either be started a fixed number of days after having passed the white blood cell nadir (fixed-day scheme) or be based on monitoring of CD34+ cells. This study was conducted to compare both approaches and to assess possible financial consequences. STUDY DESIGN AND METHODS: For 29 patients daily enumeration of CD34+ cells was used to guide leukapheresis timing. In a retrospective analysis for the same group of patients, application of a fixed-day scheme was assumed. For scenarios of beginning apheresis 2, 3, 4, or 5 days after WBC nadir, the number of apheresis days and granulocyte-colony-stimulating factor (G-CSF) application days that could be saved was calculated. RESULTS: A total of 44 apheresis procedures were performed resulting in a mean CD34+ cell content per apheresis product of 10.4 x 10(6) (range, 0.1 x 10(6)-49.5 x 10(6))/kg of body weight. The smallest number of deviation days compared to a fixed-day scheme was found for beginning an apheresis on Day 3. In comparison to this, CD34+ monitoring reduced the number of G-CSF days by 9 and the number of apheresis procedures by 11 overall, resulting in savings of euro;19,965 (US$28,788) in comparison to expenses of euro826 (US$1191) for CD34+ monitoring. CONCLUSIONS: Measurement of CD34+ cells has reached a precision enabling a prediction of the harvest success. In comparison to a fixed-day scheme, daily CD34+ monitoring reduces the donor's exposition to G-CSF, enables collection of a sufficient number of PBPCs in the least possible number of apheresis sessions, and improves the economic efficacy of the institution.


Subject(s)
Antigens, CD34 , Blood Component Removal/economics , Hematopoietic Stem Cell Mobilization/economics , Hematopoietic Stem Cells , Monitoring, Physiologic/economics , Peripheral Blood Stem Cell Transplantation/economics , Adult , Blood Component Removal/methods , Female , Granulocyte Colony-Stimulating Factor/administration & dosage , Granulocyte Colony-Stimulating Factor/economics , Hematopoietic Stem Cell Mobilization/methods , Humans , Male , Middle Aged , Monitoring, Physiologic/methods , Recombinant Proteins , Time Factors , Transplantation, Autologous
18.
Pharmacoeconomics ; 27(1): 55-68, 2009.
Article in English | MEDLINE | ID: mdl-19178124

ABSTRACT

The GOELAMS 072 study showed that first-line high-dose chemotherapy (HDT) with peripheral blood stem cell transplant (PBSCT) support was superior to the standard chemotherapy regimen (cyclophosphamide, doxorubicin, vincristine and prednisone; CHOP) in adults with aggressive non-Hodgkin's lymphoma (NHL). The aim of the study was to evaluate the pharmacoeconomic profile of HDT with PBSCT support relative to standard CHOP therapy as first-line treatment in adults with aggressive NHL. We performed a cost-effectiveness analysis from the French Public Health Insurance perspective, restricted to hospital costs (euro, year 2008 values). The clinical effectiveness criterion was censured overall survival (OS) difference after a median follow-up of 4 years for the entire cohort. A total of 197 patients were included (CHOP, n = 99; HDT, n = 98). Uncertainty was assessed using non-parametric bootstrap simulations and various scenario analyses. Five-year OS did not differ significantly between groups for the entire cohort. Nevertheless, subgroup analyses appeared to be more relevant for decision making: among patients with a high-intermediate risk according to the age-adjusted International Prognostic Index (IPI), HDT yielded a significantly higher 5-year OS than CHOP (74% vs 44%; p = 0.001). Among these patients, the mean censured OS survival, adjusted for time discounting and quality of life (QOL), increased with HDT by 1.20 years (95% CI 1.19, 1.21). The cost per life-year saved with HDT was estimated as euro34 315 (95% CI 32 683, 35 947) in this subgroup. Results suggested that HDT with PBSCT support might be considered a cost-effective strategy among patients with high-intermediate-risk NHL according to the age-adjusted IPI. Its place and its cost effectiveness potential versus, or in combination with, rituximab still need further research.


Subject(s)
Antineoplastic Combined Chemotherapy Protocols/economics , Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Combined Modality Therapy/economics , Cost-Benefit Analysis , Lymphoma, Non-Hodgkin/economics , Lymphoma, Non-Hodgkin/therapy , Peripheral Blood Stem Cell Transplantation/economics , Adult , Cyclophosphamide/economics , Cyclophosphamide/therapeutic use , Doxorubicin/economics , Doxorubicin/therapeutic use , Female , Humans , Male , Prednisolone/economics , Prednisolone/therapeutic use , Randomized Controlled Trials as Topic , Time Factors , Transplantation, Autologous/economics , Vincristine/economics , Vincristine/therapeutic use
19.
Saudi Med J ; 29(10): 1484-9, 2008 Oct.
Article in English | MEDLINE | ID: mdl-18946578

ABSTRACT

OBJECTIVE: To describe the experience in setting up a bone marrow transplant program at Ain Shams University, Cairo, Egypt. METHODS: Sixteen patients were transplanted at Ain Shams University Bone Marrow Transplantation unit from March 2005 to January 2008. RESULTS: Sixteen patients were transplanted with a median age of 25 years. Indications for transplantation were chronic myeloid leukemia, acute myeloid leukemia, aplastic anemia, acute lymphoblastic leukemia, and aggressive lymphoma. Seven donors and 6 patients were positive for cytomegalovirus immunoglobulin G (IgG) antibody (Ab) pretransplant. Only one patient was positive for toxoplasma IgG Ab and another had a high titre for toxoplasma IgM Ab pretransplant. Two donors and 2 recipients were positive for hepatitis B antibody markers; however, none were positive for hepatitis B virus DNA by polymerase chain reaction (PCR). None of the patients or donors were positive for hepatitis C virus via PCR pre-transplant. Acute graft versus host disease (GVHD) was seen in 3 patients, while chronic GVHD was seen in 5 patients. Primary cause of death was recurrence in 2 patients and graft failure in one patient. Thirteen are alive and disease free with a median follow-up of 20 months. CONCLUSION: Although our unit is a relatively new unit, these results are comparable to those achieved in the Western world and cost a mean of US$250,000.


Subject(s)
Health Care Costs , Hematologic Diseases/therapy , Peripheral Blood Stem Cell Transplantation , Adolescent , Adult , Child , Child, Preschool , Egypt , Hematologic Diseases/economics , Hematopoietic Stem Cell Mobilization , Humans , Peripheral Blood Stem Cell Transplantation/adverse effects , Peripheral Blood Stem Cell Transplantation/economics , Peripheral Blood Stem Cell Transplantation/methods , Retrospective Studies , Survival Analysis , Transplantation Conditioning , Treatment Outcome
20.
J Support Oncol ; 4(4): 187-90, 2006 Apr.
Article in English | MEDLINE | ID: mdl-16669462

ABSTRACT

This study is a prospective evaluation of the time commitment and financial requirements of caregivers of autologous stem cell recipients during the period of inpatient hospitalization. Eligible patients identified one caregiver, and a one-page survey addressing the necessary time commitment and out-of-pocket expenses was completed by the caregiver at each visit. The caregivers of 40 patients participated (non-Hodgkin's lymphoma [n = 19], multiple myeloma [n = 18], Hodgkin's lymphoma [n = 2], or acute myelogenous leukemia [n = 1]). Caregivers included spouses (n = 35), partners/friends (n = 2), or family members (n = 3). Results were summarized for the patient's total length of stay. Each caregiver traveled a median of 829 miles over 17.8 hours. Out-of-pocket expenses varied greatly depending on whether a caregiver stayed in local accommodations (cohort 1; n = 11) or in the patient's hospital room (cohort 2; n = 29). Total expenses (median) for each caregiver in cohort 1 were dollar 849.35, including accommodations (dollar 560), gasoline (dollar 87.35), and food (dollar 202). Total expenses (median) for each caregiver in cohort 2 were dollar 181.15, including gasoline (dollar 70) and food (dollar 111.15). Each caregiver in cohort 1 lost a median of 43.5 hours of work compared with 8 hours for each caregiver in cohort 2. The results from this prospective study demonstrate that there is a significant financial and time requirement on the part of the caregiver when a family member or significant other is hospitalized for an autologous stem cell transplant.


Subject(s)
Caregivers/economics , Cost of Illness , Peripheral Blood Stem Cell Transplantation/economics , Adult , Aged , Caregivers/psychology , Employment , Female , Humans , Male , Middle Aged , Peripheral Blood Stem Cell Transplantation/psychology , Prospective Studies , Surveys and Questionnaires , Time , Transplantation, Autologous
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