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How I perform hematopoietic stem cell transplantation on patients with a history of invasive fungal disease. Blood 2021; 136:2741-2753. [PMID: 33301030 DOI: 10.1182/blood.2020005884] [Citation(s) in RCA: 6] [Impact Index Per Article: 1.5] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/19/2020] [Accepted: 08/13/2020] [Indexed: 02/08/2023] Open
Abstract
Hematopoietic transplantation is the preferred treatment for many patients with hematologic malignancies. Some patients may develop invasive fungal diseases (IFDs) during initial chemotherapy, which need to be considered when assessing patients for transplantation and treatment posttransplantation. Given the associated high risk of relapse and mortality in the post-hematopoietic stem cell transplantation (HSCT) period, IFDs, especially invasive mold diseases, were historically considered a contraindication for HSCT. Over the last 3 decades, advances in antifungal drugs and early diagnosis have improved IFD outcomes, and HSCT in patients with a recent IFD has become increasingly common. However, an organized approach for performing transplantation in patients with a prior IFD is scarce, and decisions are highly individualized. Patient-, malignancy-, transplantation procedure-, antifungal treatment-, and fungus-specific issues affect the risk of IFD relapse. Effective surveillance to detect IFD relapse post-HSCT and careful drug selection for antifungal prophylaxis are of paramount importance. Antifungal drugs have their own toxicities and interact with immunosuppressive drugs such as calcineurin inhibitors. Immune adjunct cytokine or cellular therapy and surgery can be considered in selected cases. In this review, we critically evaluate these factors and provide guidance for the complex decision making involved in the peri-HSCT management of these patients.
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Sampaio MM, Santos MLC, Marques HS, Gonçalves VLDS, Araújo GRL, Lopes LW, Apolonio JS, Silva CS, Santos LKDS, Cuzzuol BR, Guimarães QES, Santos MN, de Brito BB, da Silva FAF, Oliveira MV, Souza CL, de Melo FF. Chronic myeloid leukemia-from the Philadelphia chromosome to specific target drugs: A literature review. World J Clin Oncol 2021; 12:69-94. [PMID: 33680875 PMCID: PMC7918527 DOI: 10.5306/wjco.v12.i2.69] [Citation(s) in RCA: 30] [Impact Index Per Article: 7.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 08/11/2020] [Revised: 12/22/2020] [Accepted: 01/28/2021] [Indexed: 02/06/2023] Open
Abstract
Chronic myeloid leukemia (CML) is a myeloproliferative neoplasm and was the first neoplastic disease associated with a well-defined genotypic anomaly - the presence of the Philadelphia chromosome. The advances in cytogenetic and molecular assays are of great importance to the diagnosis, prognosis, treatment, and monitoring of CML. The discovery of the breakpoint cluster region (BCR)-Abelson murine leukemia (ABL) 1 fusion oncogene has revolutionized the treatment of CML patients by allowing the development of targeted drugs that inhibit the tyrosine kinase activity of the BCR-ABL oncoprotein. Tyrosine kinase inhibitors (known as TKIs) are the standard therapy for CML and greatly increase the survival rates, despite adverse effects and the odds of residual disease after discontinuation of treatment. As therapeutic alternatives, the subsequent TKIs lead to faster and deeper molecular remissions; however, with the emergence of resistance to these drugs, immunotherapy appears as an alternative, which may have a cure potential in these patients. Against this background, this article aims at providing an overview on CML clinical management and a summary on the main targeted drugs available in that context.
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Affiliation(s)
- Mariana Miranda Sampaio
- Instituto Multidisciplinar em Saúde, Universidade Federal da Bahia, Vitória da Conquista 45029-094, Bahia, Brazil
| | - Maria Luísa Cordeiro Santos
- Instituto Multidisciplinar em Saúde, Universidade Federal da Bahia, Vitória da Conquista 45029-094, Bahia, Brazil
| | - Hanna Santos Marques
- Campus Vitória da Conquista, Universidade Estadual do Sudoeste da Bahia, Vitória da Conquista 45083-900, Bahia, Brazil
| | | | - Glauber Rocha Lima Araújo
- Instituto Multidisciplinar em Saúde, Universidade Federal da Bahia, Vitória da Conquista 45029-094, Bahia, Brazil
| | - Luana Weber Lopes
- Instituto Multidisciplinar em Saúde, Universidade Federal da Bahia, Vitória da Conquista 45029-094, Bahia, Brazil
| | - Jonathan Santos Apolonio
- Instituto Multidisciplinar em Saúde, Universidade Federal da Bahia, Vitória da Conquista 45029-094, Bahia, Brazil
| | - Camilo Santana Silva
- Instituto Multidisciplinar em Saúde, Universidade Federal da Bahia, Vitória da Conquista 45029-094, Bahia, Brazil
| | - Luana Kauany de Sá Santos
- Instituto Multidisciplinar em Saúde, Universidade Federal da Bahia, Vitória da Conquista 45029-094, Bahia, Brazil
| | - Beatriz Rocha Cuzzuol
- Instituto Multidisciplinar em Saúde, Universidade Federal da Bahia, Vitória da Conquista 45029-094, Bahia, Brazil
| | | | - Mariana Novaes Santos
- Instituto Multidisciplinar em Saúde, Universidade Federal da Bahia, Vitória da Conquista 45029-094, Bahia, Brazil
| | - Breno Bittencourt de Brito
- Instituto Multidisciplinar em Saúde, Universidade Federal da Bahia, Vitória da Conquista 45029-094, Bahia, Brazil
| | | | - Márcio Vasconcelos Oliveira
- Instituto Multidisciplinar em Saúde, Universidade Federal da Bahia, Vitória da Conquista 45029-094, Bahia, Brazil
| | - Cláudio Lima Souza
- Instituto Multidisciplinar em Saúde, Universidade Federal da Bahia, Vitória da Conquista 45029-094, Bahia, Brazil
| | - Fabrício Freire de Melo
- Instituto Multidisciplinar em Saúde, Universidade Federal da Bahia, Vitória da Conquista 45029-094, Bahia, Brazil
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Kanate AS, Majhail NS, Savani BN, Bredeson C, Champlin RE, Crawford S, Giralt SA, LeMaistre CF, Marks DI, Omel JL, Orchard PJ, Palmer J, Saber W, Veys PA, Carpenter PA, Hamadani M. Indications for Hematopoietic Cell Transplantation and Immune Effector Cell Therapy: Guidelines from the American Society for Transplantation and Cellular Therapy. Biol Blood Marrow Transplant 2020; 26:1247-1256. [PMID: 32165328 DOI: 10.1016/j.bbmt.2020.03.002] [Citation(s) in RCA: 157] [Impact Index Per Article: 31.4] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/29/2020] [Accepted: 03/02/2020] [Indexed: 12/20/2022]
Abstract
The American Society for Transplantation and Cellular Therapy (ASTCT) published its first white paper on indications for autologous and allogeneic hematopoietic cell transplantation (HCT) in 2015. It was identified at the time that periodic updates of indications would be required to stay abreast with state of the art and emerging indications and therapy. In recent years the field has not only seen an improvement in transplantation technology, thus widening the therapeutic scope of HCT, but additionally a whole new treatment strategy using modified immune effector cells, including chimeric antigen receptor T cells and engineered T-cell receptors, has emerged. The guidelines review committee of the ASTCT deemed it optimal to update the ASTCT recommendations for indications for HCT to include new data and to incorporate indications for immune effector cell therapy (IECT) where appropriate. The guidelines committee established a multiple stakeholder task force consisting of transplant experts, payer representatives, and a patient advocate to provide guidance on indications for HCT and IECT. This article presents the updated recommendations from the ASTCT on indications for HCT and IECT. Indications for HCT/IECT were categorized as (1) Standard of care, where indication is well defined and supported by evidence; (2) Standard of care, clinical evidence available, where large clinical trials and observational studies are not available but have been shown to be effective therapy; (3) Standard of care, rare indication, for rare diseases where demonstrated effectiveness exists but large clinical trials and observational studies are not feasible; (4) Developmental, for diseases where preclinical and/or early-phase clinical studies show HCT/IECT to be a promising treatment option; and (5) Not generally recommended, where available evidence does not support the routine use of HCT/IECT. The ASTCT will continue to periodically review these guidelines and update them as new evidence becomes available.
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Affiliation(s)
- Abraham S Kanate
- Hematopoietic Malignancy & Cellular Therapy Program, West Virginia University, Morgantown, West Virginia.
| | - Navneet S Majhail
- Blood & Marrow Transplant Program, Cleveland Clinic, Cleveland, Ohio
| | - Bipin N Savani
- Division of Hematology/Oncology, Vanderbilt University Medical Center, Nashville, Tennessee
| | - Christopher Bredeson
- Division of Hematology, University of Ottawa and Ottawa Hospital Research Institute, Ottawa, Ontario, Canada
| | - Richard E Champlin
- Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas
| | | | - Sergio A Giralt
- Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, New York
| | | | - David I Marks
- Adult BMT Unit, University Hospitals Bristol NHS Foundation Trust, Bristol, UK
| | | | - Paul J Orchard
- Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota
| | - Jeanne Palmer
- Division of Hematology/Oncology, Mayo Clinic, Phoenix, Arizona
| | - Wael Saber
- BMT & Cellular Therapy Program, Medical College of Wisconsin, Milwaukee, Wisconsin; Center for International Blood and Marrow Transplant Research, Milwaukee, Wisconsin
| | - Paul A Veys
- Bone Marrow Transplantation Unit, Great Ormond Street Hospital for Children, London, UK
| | - Paul A Carpenter
- Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington
| | - Mehdi Hamadani
- BMT & Cellular Therapy Program, Medical College of Wisconsin, Milwaukee, Wisconsin; Center for International Blood and Marrow Transplant Research, Milwaukee, Wisconsin
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Hoogland AI, Nelson AM, Small BJ, Hyland KA, Gonzalez BD, Booth-Jones M, Anasetti C, Jacobsen PB, Jim HSL. The Role of Age in Neurocognitive Functioning among Adult Allogeneic Hematopoietic Cell Transplant Recipients. Biol Blood Marrow Transplant 2017; 23:1974-1979. [PMID: 28797784 DOI: 10.1016/j.bbmt.2017.08.006] [Citation(s) in RCA: 21] [Impact Index Per Article: 2.6] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/17/2017] [Accepted: 08/02/2017] [Indexed: 01/02/2023]
Abstract
Improvements in supportive care have enabled allogeneic hematopoietic cell transplantation (HCT) to be performed in increasingly older patients. HCT is associated with neurocognitive impairment, which may be exacerbated in older adults due to normal neurocognitive decline associated with aging. The goal of this study was to evaluate whether increasing age of allogeneic HCT recipients is associated with worse neurocognitive outcomes over time relative to a matched sample of individuals without cancer. Patients (n = 140; 42% female; M age, 51 years; range, 20 to 76 years; 31% with acute myelogenous leukemia) completed neurocognitive assessments before transplantation and 3 months and 1 year after transplantation. Controls (n = 75; 56% female; M age, 53 years; range, 21 to 74 years) completed assessments at comparable time intervals. Linear mixed models revealed that regardless of age, patients demonstrated worse performance than controls before transplantation in verbal memory, visual memory, and total neuropsychological performance, and over time in executive functioning. In addition, older age was associated with worse performance in verbal memory (P = .02) and verbal fluency (P = .05) over time in patients compared with controls. Specifically, older (65+ years) patients had worse verbal memory and verbal fluency than older and younger (<65 years) controls post-transplantation (Cohen's d = .22 to .39). These data indicate that age may be a risk factor for worse neurocognitive outcomes after allogeneic HCT. If replicated, our results suggest that older candidates for allogeneic HCT should be counseled regarding the risk of cognitive problems after transplantation.
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Affiliation(s)
- Aasha I Hoogland
- Department of Health Outcomes and Behavior, Moffitt Cancer Center, Tampa, Florida; Department of Oncologic Sciences, Morsani College of Medicine, University of South Florida, Tampa, Florida
| | - Ashley M Nelson
- Department of Psychology, University of South Florida, Tampa, Florida
| | - Brent J Small
- School of Aging Studies, University of South Florida, Tampa, Florida
| | - Kelly A Hyland
- Department of Psychology, University of South Florida, Tampa, Florida
| | - Brian D Gonzalez
- Department of Health Outcomes and Behavior, Moffitt Cancer Center, Tampa, Florida
| | - Margaret Booth-Jones
- Department of Health Outcomes and Behavior, Moffitt Cancer Center, Tampa, Florida
| | - Claudio Anasetti
- Department of Health Outcomes and Behavior, Moffitt Cancer Center, Tampa, Florida
| | - Paul B Jacobsen
- Healthcare Delivery Research Program, National Cancer Institute, Bethesda, Maryland
| | - Heather S L Jim
- Department of Health Outcomes and Behavior, Moffitt Cancer Center, Tampa, Florida.
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Majhail NS, Farnia SH, Carpenter PA, Champlin RE, Crawford S, Marks DI, Omel JL, Orchard PJ, Palmer J, Saber W, Savani BN, Veys PA, Bredeson CN, Giralt SA, LeMaistre CF. Indications for Autologous and Allogeneic Hematopoietic Cell Transplantation: Guidelines from the American Society for Blood and Marrow Transplantation. Biol Blood Marrow Transplant 2015; 21:1863-1869. [PMID: 26256941 DOI: 10.1016/j.bbmt.2015.07.032] [Citation(s) in RCA: 285] [Impact Index Per Article: 28.5] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 07/30/2015] [Accepted: 07/31/2015] [Indexed: 02/07/2023]
Abstract
Approximately 20,000 hematopoietic cell transplantation (HCT) procedures are performed in the United States annually. With advances in transplantation technology and supportive care practices, HCT has become safer, and patient survival continues to improve over time. Indications for HCT continue to evolve as research refines the role for HCT in established indications and identifies emerging indications where HCT may be beneficial. The American Society for Blood and Marrow Transplantation (ASBMT) established a multiple-stakeholder task force consisting of transplant experts, payer representatives, and a patient advocate to provide guidance on "routine" indications for HCT. This white paper presents the recommendations from the task force. Indications for HCT were categorized as follows: (1) Standard of care, where indication for HCT is well defined and supported by evidence; (2) Standard of care, clinical evidence available, where large clinical trials and observational studies are not available but HCT has been shown to be effective therapy; (3) Standard of care, rare indication, for rare diseases where HCT has demonstrated effectiveness but large clinical trials and observational studies are not feasible; (4) Developmental, for diseases where preclinical and/or early phase clinical studies show HCT to be a promising treatment option; and (5) Not generally recommended, where available evidence does not support the routine use of HCT. The ASBMT will periodically review these guidelines and will update them as new evidence becomes available.
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Affiliation(s)
| | | | - Paul A Carpenter
- Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA
| | - Richard E Champlin
- Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX
| | | | - David I Marks
- Adult BMT Unit, University Hospitals Bristol NHS Foundation Trust, Bristol, UK
| | | | - Paul J Orchard
- Department of Pediatrics, University of Minnesota, Minneapolis, MN
| | - Jeanne Palmer
- Division of Hematology/Oncology, Mayo Clinic, Phoenix, AZ
| | - Wael Saber
- Division of Hematology and Oncology, Medical College of Wisconsin, Milwaukee, WI
- Center for International Blood and Marrow Transplant Research, Milwaukee, WI
| | - Bipin N Savani
- Division of Hematology/Oncology, Vanderbilt University Medical Center, Nashville, TN
| | - Paul A Veys
- Bone Marrow Transplantation Unit, Great Ormond Street Hospital for Children, London, UK
| | | | - Sergio A Giralt
- Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY
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Costa LJ, Huang JX, Hari PN. Disparities in utilization of autologous hematopoietic cell transplantation for treatment of multiple myeloma. Biol Blood Marrow Transplant 2014; 21:701-6. [PMID: 25555447 DOI: 10.1016/j.bbmt.2014.12.024] [Citation(s) in RCA: 60] [Impact Index Per Article: 5.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/16/2014] [Accepted: 12/19/2014] [Indexed: 12/22/2022]
Abstract
Autologous hematopoietic cell transplantation (AHCT) is an established therapy for multiple myeloma (MM), with an impact on quality of remission and survival. We analyzed the role of race, ethnicity, sex, and age disparities in AHCT utilization in the United States. We combined MM incidence derived from the Surveillance, Epidemiology and End Results program with transplantation activity reported to the Center for International Blood and Marrow Transplant Research for the period of 2005 to 2009 to assess the impact of disparities in AHCT. Utilization (number of transplantations/new cases) was compared between groups using the relative utilization ratio (RUR), defined as [utilization for a given category]/[utilization for the entire population]. Data were obtained from 22,462 actual MM cases and 13,311 AHCT. The age-adjusted RUR was 1.17 (95% confidence interval [CI], 1.15 to 1.19) among non-Hispanic Whites (NHW), higher than in non-Hispanic Blacks (NHB) (age-adjusted RUR, .69; 95% CI, .67 to .72; P < .0002), Hispanics (age-adjusted RUR, .64; 95% CI, .60 to .69; P < .002), and Asians (age-adjusted RUR, .65; 95% CI, .58 to .73; P < .0002]. AHCT utilization was higher in men than in women among Hispanics (age-adjusted RUR .72 versus .56, P = .007), but not among NHW, NHB, or Asians. Sex disparity prevents 1.3% of potential AHCTs in patients with MM (10.4% among Hispanics). Racial-ethnic disparities prevent 13.8% of AHCTs (44.7% in Hispanic and Asians, 39.9% in NHBs). Race-ethnicity disparity greatly affects AHCT utilization in MM. Sex disparity plays a lesser role, except among Hispanics. The ongoing decrease in age disparity will continue to drive major increase of AHCT activity. Two-year and 5-year increases in the age of the AHCT population would result in 12% and 32% increases, respectively, in volume of AHCT.
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Affiliation(s)
- Luciano J Costa
- Division of Hematology and Oncology, Department of Medicine, University of Alabama at Birmingham, Birmingham, Alabama.
| | - Jia-Xing Huang
- Center for International Blood and Marrow Transplant Research, Milwaukee, Wisconsin
| | - Parameswaran N Hari
- Center for International Blood and Marrow Transplant Research, Milwaukee, Wisconsin; Division of Hematology and Oncology, Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin
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