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

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.

Report 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.
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ABOUT DATAMONITOR HEALTHCARE 2
About the Oncology pharmaceutical analysis team 2
CHAPTER 1 EXECUTIVE SUMMARY 3
Scope of analysis 3
Datamonitor insight into hematopoietic stem cell transplantation 4
Contributing experts 5
Related reports 5
Upcoming reports 6
CHAPTER 2 HEMATOPOIETIC STEM CELL TRANSPLANTATION OVERVIEW 8
Definition 8
Overview of the hematopoietic stem cell transplantation procedure 9
Use and applications of hematopoietic stem cell transplantation 11
Types of stem cell transplants 15
Stem cell donors 15
Allogeneic transplants 15
Autologous transplants 18
Stem cell sources 18
Bone marrow 20
Peripheral blood 21
Umbilical cord blood 22
Conditioning 23
Myeloablative conditioning 23
Reduced-intensity conditioning (RIC) and nonmyeloablative conditioning 24
Mortality and survival data 28
CHAPTER 3 PHARMACOLOGIC USE 30
Conditioning regimens 30
Myeloablative regimens 30
Reduced-intensity conditioning 31
Conditioning by type of hematologic malignancy 32
Research & development in the conditioning field 33
Molecular targeted therapies 33
Radioimmunotherapy 35
Prophylaxis and treatment of hematopoietic stem cell transplantation complications 36
Early complications 36
Acute graft-versus-host disease (GVHD) 38
Late complications 42
Chronic graft-versus-host disease (GVHD) 43
Secondary malignancies 45
Other areas of research & development 45
Augmentation of the graft-versus-tumor (GVT) effect 45
Expansion of allogeneic HSCT to solid tumors 46
CHAPTER 4 UNMET NEEDS & RESEARCH PRIORITIES 47
Key findings 47
Unmet needs and research priorities in hematopoietic stem cell transplantation 47
HSCT remains an underutilized treatment 47
Relapse remains the main cause of death after HSCT 49
HSCT is in need of an overall refinement 49
The effective management of graft-versus-host disease is considered a priority 50
BIBLIOGRAPHY 52
APPENDIX 62
List of tables 62
List of figures 62
About Datamonitor 62
About Datamonitor Healthcare 62
About the Oncology analysis team 63
Disclaimer 66
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Table 1: Hematopoietic stem cell transplantation: definitions 8
Table 2: Donor types for allogeneic hematopoietic stem cell transplantation 16
Table 3: Comparison of stem cell sources for hematopoietic stem cell transplantation 20
Table 4: Reduced-intensity conditioning (RIC) and nonmyeloablative regimens: mode of action 24
Table 5: Three-year survival rates for patients with acute myeloid leukemia and diffuse large B-cell lymphoma 29
Table 6: Examples of radiation-based conditioning regimens used in hematopoietic stem cell transplantation 30
Table 7: Examples of chemotherapy-based conditioning regimens used in hematopoietic stem cell transplantation 31
Table 8: Examples of reduced-intensity conditioning regimens used in hematopoietic stem cell transplantation 31
Table 9: Examples of conditioning regimens used in hematopoietic stem cell transplantation by type of hematologic malignancy 32
Table 10: Investigation of Rituxan and Campath in regimens used in hematopoietic stem cell transplantation conditioning 33
Table 11: Investigation of anti-CD20 radioimmunotherapy in hematopoietic stem cell transplantation conditioning 36
Table 12: Early complications of hematopoietic stem cell transplantation 37
Table 13: Selected drugs available for use in the prophylaxis of acute graft-versus-host disease 38
Table 14: Selected drugs investigated in the treatment of steroid-refractory acute graft-versus-host disease 40
Table 15: Drugs/therapies in company-sponsored Phase III development for graft-versus-host disease as their primary indication, 2009 41
Table 16: Late complications of hematopoietic stem cell transplantation 42
Table 17: Selected drugs investigated in the treatment of steroid-refractory chronic graft-versus-host disease 44
Figure 1: The normal hematopoiesis process 9
Figure 2: Overview of the hematopoietic stem cell transplantation procedure 10
Figure 3: The applications of autologous and allogeneic hematopoietic stem cell transplantation 11
Figure 4: Main indications for hematopoietic stem cell transplantation in 2007, according to EBMT data 12
Figure 5: Indications for allogeneic hematopoietic stem cell transplantation worldwide in 2005, according to CIBMTR data 13
Figure 6: Annual number of hematopoietic stem cell transplantations worldwide, 1970-2006, according to CIBMTR data 14
Figure 7: Stem cell sources for allogeneic and autologous transplants in patients over 20 years of age, 1997-2006, according to CIBMTR data 19
Figure 8: The use of myeloablative and reduced-intensity conditioning regimens in allogeneic transplants, 1998-2006 according to CIBMTR data 26
Figure 9: Age distribution of patients receiving allogeneic transplants by conditioning regimen intensity, 2005-06, according to CIBMTR data 27
Figure 10: Causes of death after transplantations, 2001-06, according to CIBMTR data 28
Figure 11: Unmet needs and research priorities in hematopoietic stem cell transplantation 47
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