Abstract:
:In the United States the increasing number of Food and Drug Administration (FDA)-approved, innovative, and potentially effective commercial cancer therapies pose a significant financial burden on public and private payers. Chimeric antigen receptor (CAR) T cells are prototypical of this challenge. In 2017 and 2018, tisagenlecleucel (Kymriah, Novartis) and axicabtagene ciloleucel (Yescarta, Kite) were approved by the FDA for use after showing groundbreaking results in relapsed/refractory B-cell malignancies. In 2020 and 2021, four further submissions to the FDA are expected for CAR T-cell therapies for indolent and aggressive B-cell malignancies and plasma cell myeloma. Yet, with marketed prices of over $350,000 per infusion for the two FDA-approved therapies and similar price tags expected for the coming products, serious concerns are raised over value and affordability. In this review we summarize recent, peer-reviewed cost-effectiveness studies of tisagenlecleucel and axicabtagene ciloleucel in the United States; discuss key issues concerning the health plan budget impact of CAR T-cell therapy; and review policy, payment and scientific approaches that may improve the value and affordability of CAR T-cell therapy.
journal_name
Bone Marrow Transplantjournal_title
Bone marrow transplantationauthors
Fiorenza S,Ritchie DS,Ramsey SD,Turtle CJ,Roth JAdoi
10.1038/s41409-020-0956-8subject
Has Abstractpub_date
2020-09-01 00:00:00pages
1706-1715issue
9eissn
0268-3369issn
1476-5365pii
10.1038/s41409-020-0956-8journal_volume
55pub_type
杂志文章,评审abstract::Safety of living donors is critical to the success of blood, tissue and organ transplantation. Structured and robust vigilance and surveillance systems exist as part of some national entities, but historically no global systems are in place to ensure conformity, harmonisation and the recognition of rare adverse events...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2013.104
更新日期:2013-11-01 00:00:00
abstract::To find an approach to prolong the survival time of cardiac allografts in a BALB/c-to-C57/BL6 heterotopic heart transplant model and to try to figure out related chemokines and cytokines, isogeneic and allogeneic BM cells were obtained from pregnant C57/BL6 (♀C57/BL6 × ♂BALB/c) and regular BALB/c mice and injected to ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2011.215
更新日期:2012-08-01 00:00:00
abstract::Sequential administration of high-dose chemotherapy courses possibly allows extensive in vivo purging before circulating progenitor collection for autograft. To evaluate whether progenitor cell mobilization was negatively affected by repeated high-dose chemotherapy courses, we studied 23 lymphoma patients undergoing t...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-08-01 00:00:00
abstract::Although infants with stage 4 neuroblastoma (NB) usually have a good prognosis, metastatic relapses after 1 year of age and amplification of the N-myc oncogene are established poor prognostic factors. In order to improve the survival of patients with such high-risk factors, we performed consolidation with a busulfan (...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1702376
更新日期:2000-05-01 00:00:00
abstract::The potent immunostimulatory cytokine interleukin-2 (IL-2) has been extensively investigated for its potential to induce anti-tumor immunity in a number of tumor models. Only recently the complex interplay of mutually suppressive or supportive cytokines of the IL-2-induced network of cytokines has been better characte...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1702431
更新日期:2000-07-01 00:00:00
abstract::The homing of hematopoietic precursor cells (HPC) within the bone marrow is most likely to be mediated by specific adhesion via surface receptors to cellular and extracellular matrix (ECM) components and to be regulated by cytokines. We investigated the effects of serum and cytokines on the expression of adhesion mole...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701484
更新日期:1998-12-01 00:00:00
abstract::The interaction between CXCL12 and its receptor CXCR4 plays a crucial role in the homing and mobilization of haematopoietic progenitors. We investigated the putative association between a CXCL12 gene polymorphism, the G --> A transition at position 801 in the 3'-untranslated region (3'UTR), and the yield of CD34(+) pr...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2009.30
更新日期:2009-09-01 00:00:00
abstract::Allogeneic hematopoietic stem cell transplantation (SCT) from unaffected donors remains the only modality for the correction of hematological abnormalities in Fanconi anemia (FA) patients. We performed four HLA-matched related donor SCT using a novel irradiation and cyclophosphamide-free conditioning regimen. The prot...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704580
更新日期:2004-08-01 00:00:00
abstract::Seventy consecutive patients with refractory or relapsed Hodgkin's disease who received high-dose chemotherapy followed by autologous stem cell rescue were analyzed to identify clinically relevant predictors of long-term event-free survival. High-dose therapy consisted primarily of carmustine (BCNU), etoposide, cytara...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701325
更新日期:1998-08-01 00:00:00
abstract::Patterns of C-reactive protein (CRP) release were derived from frequent CRP measurements in a cohort of 66 consecutive patients receiving allogeneic bone marrow transplants (BMT) in our unit. Based on a retrospective study of clinical events occurring within the first 40 days after BMT, patients with major transplant-...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701286
更新日期:1998-07-01 00:00:00
abstract::Allogeneic hematopoietic stem cell transplantation (allo-HSCT) has proven effective in adult T-cell leukemia/lymphoma (ATL) patients. To study the graft-versus-ATL (Gv-ATL) effects after allo-HSCT, we analyzed 21 ATL patients who had been treated at our hospital. Of these, 18 had acute-, 2 had lymphoma- and 1 had chro...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2008.39
更新日期:2008-06-01 00:00:00
abstract::Information regarding the chimeric status of hematopoietic stem cell transplantation (HSCT) recipients is of great significance when comparing different conditioning and prophylactic therapies. In recent years, short tandem repeats/variable number tandem repeats (STRs/VNTRs) have emerged as the best tool for chimerism...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,随机对照试验
doi:10.1038/sj.bmt.1705626
更新日期:2007-05-01 00:00:00
abstract::Respiratory syncytial virus (RSV), a common cause of both upper and lower respiratory tract infection (LRTI) in infants and children, is rarely described as an infective agent in adults. It has been reported in bone marrow transplant (BMT) recipients and patients with malignancy immunosuppressed by chemotherapy. Such ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1701091
更新日期:1998-02-01 00:00:00
abstract::We examined whether pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF) is capable of improving thrombocytopenia and promoting thrombopoietic reconstitution following lethal irradiation and bone marrow transplantation (BMT) in mice. Immediately after receiving 10 Gy whole body irradia...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-12-01 00:00:00
abstract::IL-7 and IL-4 are known to influence the growth of cells of the lymphoid lineage. In this study, we investigated the effects of in vivo administration of IL-7 or IL-4 in mice subjected to congenic BM transplant. C57BL/6 Ly5.1+ mice were subjected to TBI, followed by transfer of B and T cell-depleted BM from C57BL/6 Ly...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1995-07-01 00:00:00
abstract::In the original version of this article, the mention of 'ifosfamide 1500 mg/m2 days 1-3' should, in fact, read 'ifosfamide 1500 mg/m2 bd days 1-3'. This has now been updated in the original version of the article. ...
journal_title:Bone marrow transplantation
pub_type: 已发布勘误
doi:10.1038/s41409-019-0504-6
更新日期:2020-04-01 00:00:00
abstract::The toxicity of the conditioning regimen high-dose busulfan (BU) 16 mg/kg followed by cyclophosphamide (CY) 200 mg/kg has been analysed in 60 adult patients (mean age 36 +/- 9 years) with haematological malignancies, a third of whom had advanced disease, all received the graft from fully HLA-identical siblings. Signif...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1994-05-01 00:00:00
abstract::Ocular problems are common in recipients of stem cell transplantation (SCT), but ptosis is rarely reported and investigated. Among 346 consecutive SCT recipients, severe bilateral ptosis was noticed or reported in six cases (five women and one man), all with acute graft-versus-host disease (aGVHD) and chronic graft-ve...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704608
更新日期:2004-09-01 00:00:00
abstract::Transplantation-associated thrombotic microangiopathy (TA-TMA) is a feared complication of allogeneic hematopoietic SCT (HSCT) owing to its high mortality rate. The use of calcineurin inhibitors or sirolimus (SIR) for GVHD prophylaxis has been suggested as a potential risk factor. However, the impact of tacrolimus (TA...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/bmt.2014.17
更新日期:2014-05-01 00:00:00
abstract::Corticosteroid-resistant GVHD is difficult to manage and is associated with high morbidity and mortality. Cyclophosphamide (Cy) is an established immunosuppressive and cytotoxic drug widely used as part of pretransplant conditioning regimens. In a retrospective study of 15 patients who had not responded to corticoster...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704829
更新日期:2005-04-01 00:00:00
abstract::We have evaluated bone marrow morphology, percentage of bone marrow CD34(+) cells, proliferative activity of bone marrow precursors, clonogenic assay (BFU-E and CFU-GM) in short-term bone marrow cultures, and bone marrow cell apoptosis, together with serum TNF-alpha and IL-6, in 16 chronic, refractory RA patients, as ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704407
更新日期:2004-04-01 00:00:00
abstract:SUMMARY:Hematopoietic chimerism (HpC) was assayed monthly using a sensitive, polymerase chain reaction (PCR) -based method in consecutive patients. Between January 1998 and April 2002, 181 patients underwent non-T cell depleted allogeneic hematopoietic cell transplantation (HCT). A total of 163 patients were evaluable ...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:10.1038/sj.bmt.1704147
更新日期:2003-08-01 00:00:00
abstract::The bare lymphocyte syndrome is a combined immunodeficiency resulting from the lack of expression of either class I or class II HLA antigens at the cell surface. The main clinical manifestations are infections of the respiratory or the digestive tract. The immunodeficiency involves the absence of antibody formation an...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:
更新日期:1992-01-01 00:00:00
abstract::C3H/HeJ mice were exposed to 8 or 9 Gy total body irradiation prior to transplantation of 1-15 x 10(6) H-2 incompatible T cell-depleted bone marrow cells from C57BL/6 donors. Survival was greatly enhanced compared to irradiated controls, even at the lowest cell doses. Analysis of spleen cells 1-7 weeks post-transplant...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1988-03-01 00:00:00
abstract::Ten patients with Ph+ chronic myeloid leukemia (CML) were treated with idarubicin, cytarabine and etoposide followed by G-CSF to harvest Ph-negative progenitor cells. Six were in first chronic phase (CP1), and four beyond CP1. Between two and six aphereses (median 3, total 36) were performed starting 9-26 days (median...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-01-01 00:00:00
abstract::Bone marrow transplant-associated thrombotic microangiopathy (BMT-TM) ranges in severity from a self-limiting to a fatal disorder. There is no specific therapy for this condition to date. We have previously described a simple clinical grading system (grade 0-4) for BMT-TM; patients with grade 3-4 BMT-TM do poorly. A p...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1996-01-01 00:00:00
abstract::Pretransplant conditioning regimens designed to decrease leukemia relapse and increase leukemia-free survival in allotransplants in acute myelogenous leukemia (AML) were developed recently. We review studies of new regimens containing drugs like busulfan, cytarabine, melphalan or etoposide as well as novel radiation s...
journal_title:Bone marrow transplantation
pub_type: 杂志文章,评审
doi:
更新日期:1991-04-01 00:00:00
abstract::Graft-versus-tumour reactions as a form of adoptive immunotherapy may help prevent the recurrence of haematological malignancy following allogeneic BMT. We hypothesised that such reactions may be maximised by shortening the duration of post-transplant immunosuppression by a rapid taper of cyclosporine (CYA). CYA dose ...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章
doi:10.1038/sj.bmt.1700961
更新日期:1997-11-01 00:00:00
abstract::The effects of in vitro radiation and exposure to 4-hydroperoxycyclophosphamide (4-HC) on the production of G- and GM-CSF by different components of the human hematopoietic microenvironment are described. The marrow microenvironment is composed of fibroblasts, endothelial cells, macrophages, and adipocytes. To study t...
journal_title:Bone marrow transplantation
pub_type: 杂志文章
doi:
更新日期:1992-12-01 00:00:00
abstract::Allo-SCT using unrelated donors is a curative treatment for patients with hematological disorders. The best donor is one matched for 10/10 HLA alleles, however studies have shown an additional survival benefit when considering other genetic factors. It has been shown that a six-nucleotide insertion/deletion polymorphi...
journal_title:Bone marrow transplantation
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1038/bmt.2014.238
更新日期:2015-02-01 00:00:00