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

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