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Stakeholder Opinions: Hematopoietic Stem Cell Transplantation - In need of further research and development
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Datamonitor |
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2009³â 04¿ù |
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85285 |
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68 pages |
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Abstract
Introduction
Hematopoietic stem cell transplantation (HSCT) has evolved considerably over
the past 50 years, driven by developments in stem cell sources, donor
identification and conditioning regimens. Nowadays, it has a range of
applications, including the treatment of certain hematologic malignancies.
However, further research efforts are required to expand its use and improve
patient outcomes.
Scope of this research
- Usage data for HSCT and overview of the procedure in terms of stem cell
donors, stem cell sources, and conditioning regimens
- Discussion of the use of drug therapy in HSCT conditioning and in the
management of complications, including current research and development trends
- Unmet needs and research priorities in the field of HSCT
- Stakeholder opinions based on qualitative interviews with key opinion
leaders from the US and EU
Research and analysis highlights
The use of HSCT has been increasing in the past decades, driven by
developments in stem cell sources and conditioning regimens. However, it seems
to remain an underutilized treatment, with many patients being referred for a
transplant only at advanced stages of disease, where cure is less likely.
The development of reduced-intensity conditioning (RIC) regimens is one of the
major advances in the field of HSCT. Current research evaluating monoclonal
antibodies and radioimmunotherapy may help increase the efficacy and
specificity of conditioning therapy.
The effective management of graft-versus-host disease (GVHD) is an unmet need
in the field of HSCT. The availability of better preclinical models,
prognostic tools, and effective therapies are considered R&D priorities.
Monoclonal antibodies may have a role in the treatment of GVHD but further
research is required to investigate their potential.
Key reasons to purchase this research
- Obtain analysis of current HSCT usage data and trends in the use of stem
cell donors, stem cell sources, and conditioning regimens
- Obtain an overview of current drug use in HSCT conditioning and in the
management of HSCT complications, including graft-versus-host disease
- Identify unmet needs and research priorities in the field of HSCT
Table of Contents
EXECUTIVE SUMMARY
- Scope of analysis
- Datamonitor insight into hematopoietic stem cell transplantation
- Contributing experts
- Related reports
- Upcoming reports
HEMATOPOIETIC STEM CELL TRANSPLANTATION OVERVIEW
- Definition
- Overview of the hematopoietic stem cell transplantation procedure
- Use and applications of hematopoietic stem cell transplantation
- Types of stem cell transplants
- Stem cell donors
- Stem cell sources
- Conditioning
- Mortality and survival data
PHARMACOLOGIC USE
- Conditioning regimens
- Myeloablative regimens
- Reduced-intensity conditioning
- Conditioning by type of hematologic malignancy
- Research & development in the conditioning field
- Prophylaxis and treatment of hematopoietic stem cell transplantation
complications
- Early complications
- Late complications
- Other areas of research & development
- Augmentation of the graft-versus-tumor (GVT) effect
- Expansion of allogeneic HSCT to solid tumors
UNMET NEEDS & RESEARCH PRIORITIES
- Key findings
- Unmet needs and research priorities in hematopoietic stem cell
transplantation
- HSCT remains an underutilized treatment
- Relapse remains the main cause of death after HSCT
- HSCT is in need of an overall refinement
- The effective management of graft-versus-host disease is considered a
priority
BIBLIOGRAPHY
APPENDIX
TABLES
- Table: Hematopoietic stem cell transplantation: definitions
- Table: Donor types for allogeneic hematopoietic stem cell transplantation
- Table: Comparison of stem cell sources for hematopoietic stem cell
transplantation
- Table: Reduced-intensity conditioning (RIC) and nonmyeloablative regimens:
mode of action
- Table: Three-year survival rates for patients with acute myeloid leukemia
and diffuse large B-cell lymphoma
- Table: Examples of radiation-based conditioning regimens used in
hematopoietic stem cell transplantation
- Table: Examples of chemotherapy-based conditioning regimens used in
hematopoietic stem cell transplantation
- Table: Examples of reduced-intensity conditioning regimens used in
hematopoietic stem cell transplantation
- Table: Examples of conditioning regimens used in hematopoietic stem cell
transplantation by type of hematologic malignancy
- Table: Investigation of Rituxan and Campath in regimens used in
hematopoietic stem cell transplantation conditioning
- Table: Investigation of anti-CD20 radioimmunotherapy in hematopoietic stem
cell transplantation conditioning
- Table: Early complications of hematopoietic stem cell transplantation
- Table: Selected drugs available for use in the prophylaxis of acute
graft-versus-host disease
- Table: Selected drugs investigated in the treatment of steroid-refractory
acute graft-versus-host disease
- Table: Drugs/therapies in company-sponsored Phase III development for
graft-versus-host disease as their primary indication, 2009
- Table: Late complications of hematopoietic stem cell transplantation
- Table: Selected drugs investigated in the treatment of steroid-refractory
chronic graft-versus-host disease
FIGURES
- Figure: The normal hematopoiesis process
- Figure: Overview of the hematopoietic stem cell transplantation procedure
- Figure: The applications of autologous and allogeneic hematopoietic stem
cell transplantation
- Figure: Main indications for hematopoietic stem cell transplantation in
2007, according to EBMT data
- Figure: Indications for allogeneic hematopoietic stem cell transplantation
worldwide in 2005, according to CIBMTR data
- Figure: Annual number of hematopoietic stem cell transplantations
worldwide, 1970 2006, according to CIBMTR data
- Figure: Stem cell sources for allogeneic and autologous transplants in
patients over 20 years of age, 1997 2006, according to CIBMTR data
- Figure: The use of myeloablative and reduced-intensity conditioning
regimens in allogeneic transplants, 1998 2006 according to CIBMTR data
- Figure: Age distribution of patients receiving allogeneic transplants by
conditioning regimen intensity, 2005 06, according to CIBMTR data
- Figure: Causes of death after transplantations, 2001 06, according to
CIBMTR data
- Figure: Unmet needs and research priorities in hematopoietic stem cell
transplantation
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