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Stakeholder Opinions: Hematopoietic Stem Cell Transplantation - In need of further research and development

Published By: Datamonitor
April 2009
R313-50617
Online Download   $3,800.00
Global Site License   $9,500.00
Description

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
  • 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
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.

Reasons to Purchase
  • 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
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Table of Contents
ABOUT DATAMONITOR HEALTHCARE
About the Oncology pharmaceutical analysis team
CHAPTER 1 EXECUTIVE SUMMARY
Scope of analysis
Datamonitor insight into hematopoietic stem cell transplantation
Contributing experts
Related reports
Upcoming reports
CHAPTER 2 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
Allogeneic transplants
Autologous transplants
Stem cell sources
Bone marrow
Peripheral blood
Umbilical cord blood
Conditioning
Myeloablative conditioning
Reduced-intensity conditioning (RIC) and nonmyeloablative conditioning
Mortality and survival data
CHAPTER 3 PHARMACOLOGIC USE
Conditioning regimens
Myeloablative regimens
Reduced-intensity conditioning
Conditioning by type of hematologic malignancy
Research & development in the conditioning field
Molecular targeted therapies
Radioimmunotherapy
Prophylaxis and treatment of hematopoietic stem cell transplantation complications
Early complications
Acute graft-versus-host disease (GVHD)
Late complications
Chronic graft-versus-host disease (GVHD)
Secondary malignancies
Other areas of research & development
Augmentation of the graft-versus-tumor (GVT) effect
Expansion of allogeneic HSCT to solid tumors
CHAPTER 4 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
List of tables
List of figures
About Datamonitor
About Datamonitor Healthcare
About the Oncology analysis team
Disclaimer
List of Tables
Table 1: Hematopoietic stem cell transplantation: definitions
Table 2: Donor types for allogeneic hematopoietic stem cell transplantation
Table 3: Comparison of stem cell sources for hematopoietic stem cell transplantation
Table 4: Reduced-intensity conditioning (RIC) and nonmyeloablative regimens: mode of action
Table 5: Three-year survival rates for patients with acute myeloid leukemia and diffuse large B-cell lymphoma
Table 6: Examples of radiation-based conditioning regimens used in hematopoietic stem cell transplantation
Table 7: Examples of chemotherapy-based conditioning regimens used in hematopoietic stem cell transplantation
Table 8: Examples of reduced-intensity conditioning regimens used in hematopoietic stem cell transplantation
Table 9: Examples of conditioning regimens used in hematopoietic stem cell transplantation by type of hematologic malignancy
Table 10: Investigation of Rituxan and Campath in regimens used in hematopoietic stem cell transplantation conditioning
Table 11: Investigation of anti-CD20 radioimmunotherapy in hematopoietic stem cell transplantation conditioning
Table 12: Early complications of hematopoietic stem cell transplantation
Table 13: Selected drugs available for use in the prophylaxis of acute graft-versus-host disease
Table 14: Selected drugs investigated in the treatment of steroid-refractory acute graft-versus-host disease
Table 15: Drugs/therapies in company-sponsored Phase III development for graft-versus-host disease as their primary indication, 2009
Table 16: Late complications of hematopoietic stem cell transplantation
Table 17: Selected drugs investigated in the treatment of steroid-refractory chronic graft-versus-host disease
List of Figures
Figure 1: The normal hematopoiesis process
Figure 2: Overview of the hematopoietic stem cell transplantation procedure
Figure 3: The applications of autologous and allogeneic hematopoietic stem cell transplantation
Figure 4: Main indications for hematopoietic stem cell transplantation in 2007, according to EBMT data
Figure 5: Indications for allogeneic hematopoietic stem cell transplantation worldwide in 2005, according to CIBMTR data
Figure 6: Annual number of hematopoietic stem cell transplantations worldwide, 1970-2006, according to CIBMTR data
Figure 7: Stem cell sources for allogeneic and autologous transplants in patients over 20 years of age, 1997-2006, according to CIBMTR data
Figure 8: The use of myeloablative and reduced-intensity conditioning regimens in allogeneic transplants, 1998-2006 according to CIBMTR data
Figure 9: Age distribution of patients receiving allogeneic transplants by conditioning regimen intensity, 2005-06, according to CIBMTR data
Figure 10: Causes of death after transplantations, 2001-06, according to CIBMTR data
Figure 11: Unmet needs and research priorities in hematopoietic stem cell transplantation
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PUBLICATION:   Stakeholder Opinions: Hematopoietic Stem Cell Transplantation - In need of further research and development
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