Table of Contents
- ABOUT DATAMONITOR HEALTHCARE
- About the Oncology pharmaceutical analysis team
- CHAPTER 1 EXECUTIVE SUMMARY
- Scope of analysis
- Datamonitor insight into the MDS market
- Related reports
- Upcoming reports
- CHAPTER 2 OVERVIEW OF MYELODYSPLASTIC SYNDROMES
- Introduction
- Myelodysplastic syndromes (MDS) are a heterogeneous group of
hematological malignancies
- Risk factors in the development of MDS
- Hallmark of MDS - A hypercellular bone marrow with dysplastic changes
in combination with peripheral cytopenias
- Hypercellularity
- Dysplasia
- Peripheral cytopenias
- Mortality often results from the complications of peripheral blood
cytopenias
- MDS is often a diagnosis of exclusion
- The molecular pathogenesis of MDS
- Chromosomal abnormalities are common but little is known about the
molecular basis of the disease
- MDS arises as a multistep process
- Accelerated apoptosis underlies the cytopenias in early MDS
- Excessive proliferation accompanies disease progression
- The classification of MDS
- French American British (FAB) System
- The FAB classification system is based on morphological criteria
- Limitations of the FAB system
- World Health Organization (WHO) System
- The WHO classification system incorporates new diagnostic information
- Classification of chronic myelomonocytic leukemia (CMML)
- Limitations of the WHO classification system
- International Prognostic Scoring System (IPSS)
- The IPSS classification system is the current standard for
evaluating prognosis in MDS patients
- Limitations of the IPSS system
- Epidemiology
- The epidemiology of MDS is poorly monitored
- MDS is predominantly a disease of the elderly
- A recent study shows that the majority of patients fall into the IPSS
Low and Intermediate-1 risk groups
- The incidence of MDS is expected to rise
- There will be more than 57,000 new cases of MDS across the seven major
markets in 2016
- CHAPTER 3 CURRENT TREATMENT OPTIONS FOR MDS
- Overview of MDS management
- The aim of treatment differs in lower- and higher-risk patients
- The choice of therapy is not solely dependent on a patient' s IPSS risk
group
- Summary of current trends in MDS management
- The management of IPSS lower-risk MDS patients
- Supportive care approaches
- Red blood cell (RBC) transfusion and iron chelation therapy
- Erythropoietin (EPO)
- Granulocyte-colony stimulating factor (G-CSF)
- Platelet transfusions
- Non-chemotherapy low-intensity agents
- Thalomid (thalidomide; Celgene/Pharmion)
- Revlimid (lenalidomide; Celgene)
- Antithymocyte globulin and cyclosporin A
- Anti-TNF therapy
- Vitamin D analogs
- Low-intensity chemotherapy
- The management of IPSS higher-risk patients
- Low-intensity chemotherapy - the hypomethylating agents
- Vidaza (azacitidine; Pharmion)
- Dacogen (decitabine; MGI Pharma)
- Comparisons between Vidaza and Dacogen
- Hematopoietic stem cell transplantation
- HSCT is the only potentially curative option for MDS patients but
its use in older patients is problematic
- The morbidity and mortality risks associated with HSCT often
prohibits its use in lower-risk patients
- Different transplantation options exist for the treatment of MDS
- High-intensity chemotherapy
- High-intensity chemotherapy is an alternative option for higher-risk
patients
- The use of high-intensity chemotherapy may be difficult to justify
- The management of CMML
- Supportive care and low-intensity therapy
- High-intensity therapy
- CHAPTER 4 UNMET NEEDS IN MDS
- Summary of unmet needs
- A better understanding of the molecular pathogenesis
- Increased R&D efforts
- Low-intensity therapies with improved efficacy and better toxicity
profiles
- Low-intensity therapies to improve symptoms of anemia and quality of
life
- More effective management of thrombocytopenia
- Improved HSCT procedures
- CHAPTER 5 PIPELINE ANALYSIS
- Summary
- Pipeline overview
- Late-phase product pipeline for MDS
- Phase II product pipeline for MDS
- Phase I product pipeline for MDS
- Late-phase MDS drug profiles
- Sarasar (Lonafarnib; Schering-Plough)
- Drug overview
- Key historical events
- Clinical trial data
- Datamonitor comments
- Phase II MDS drug profiles
- MGCD0103 (Pharmion/MethylGene)
- Drug overview
- Key historical events
- Clinical trial data
- Romiplostim (Amgen)
- Drug overview
- Key historical events
- Clinical trial data
- Ceflatonin (Myelostat; ChemGenex Pharmaceuticals)
- Drug overview
- Key historical events
- Clinical trial data
- Zarnestra (Tipifarnib; Johnson & Johnson)
- Drug overview
- Key historical events
- Clinical trial data
- Cloretazine (VNP40101M; Vion Pharmaceuticals)
- Drug overview
- Key historical events
- Clinical trial data
- Clolar/Evoltra (Clofarabine; Genzyme)
- Drug overview
- Key historical events
- Clinical trial data
- Telintra (TLK-199; Telik)
- Drug overview
- Key historical events
- Clinical trial data
- APPENDIX
- Bibliography
- Key opinion leaders
- List of tables
- List of figures
- Abbreviations
- About Datamonitor
- About Datamonitor Healthcare
- About the Oncology analysis team
- Disclaimer
- List of Tables
- Table 1: Myelodysplastic syndromes (MDS)
- Table 2: Common recurring chromosomal abnormalities in MDS
- Table 3: French American British (FAB) classification of MDS
- Table 4: World Health Organization (WHO) classification of MDS
- Table 5: WHO classification of myelodysplastic/myeloproliferative
diseases (MDS/MPD)
- Table 6: Survival and progression to AML for MDS patients within the
IPSS risk groups
- Table 7: IPSS classification of MDS patients
- Table 8: MDS incidence in the seven major markets, 2002-2016
- Table 9: Treatment guidelines for MDS
- Table 10: Iron chelators available in the US and EU markets, 2007
- Table 11: Branded erythropoietins available in the US and EU markets,
2007
- Table 12: Erythropoietins: ongoing clinical trials in MDS, 2007
- Table 13: Branded colony-stimulating factors available in the US and
EU, 2007
- Table 14: Thalomid: key historical events
- Table 15: Thalomid as a single agent in MDS: clinical trial results
- Table 16: Revlimid: key historical events
- Table 17: Revlimid: ongoing clinical trials in MDS, 2007
- Table 18: Antithymocyte globulin (ATG) available in the US and EU, 2007
- Table 19: Antithymocyte globulin: ongoing clinical trials in MDS, 2007
- Table 20: Vidaza: Key historical events
- Table 21: Vidaza: ongoing clinical trials in MDS, 2007
- Table 22: Response criteria used in the Phase III study of Vidaza
versus supportive care in MDS patients
- Table 23: Dacogen: key historical events
- Table 24: Dacogen: ongoing clinical trials in MDS, 2007
- Table 25: Response criteria used in the Phase III study of Dacogen
versus supportive care in MDS patients
- Table 26: Types of hematopoietic stem cell transplantation (HSCT)
- Table 27: Comparative results of RIC HSCT studies
- Table 28: High-intensity chemotherapy regimens in MDS: clinical trial
results
- Table 29: Pipeline drugs in Phase III development for MDS, 2007
- Table 30: Pipeline drugs in Phase II development for MDS, 2007
- Table 31: Pipeline drugs in Phase I development for MDS, 2007
- Table 32: Sarasar: key historical events
- Table 33: Ongoing clinical trials involving Sarasar in MDS, 2007
- Table 34: MGCD0103: key historical events
- Table 35: Ongoing clinical trials for MGCD0103 in MDS, 2007
- Table 36: Romiplostim: key historical events
- Table 37: Ongoing clinical trials for Romiplostim in MDS, 2007
- Table 38: Ceflatonin: Key historical events
- Table 39: Zarnestra: Key historical events
- Table 40: Ongoing clinical trials involving Zarnestra in MDS, 2007
- Table 41: Final results from a Phase II study of Zarnestra in
Intermediate- and High-risk MDS
- Table 42: Cloretazine: Key historical events
- Table 43: Ongoing clinical trials involving Cloretazine in MDS, 2007
- Table 44: Clolar: Key historical events
- Table 45: Ongoing clinical trials involving Clolar in MDS, 2007
- Table 46: Telintra: key historical events
- Table 47: Ongoing clinical trials involving Telintra in MDS, 2007
- Table 48: Abbreviations used in Stakeholder Opinions: Myelodysplastic
Syndromes
- List of Figures
- Figure 1: Risk factors implicated in the onset of MDS
- Figure 2: The hematopoiesis process
- Figure 3: Hypercellularity and peripheral blood cytopenias in MDS
- Figure 4: The pathophysiology of MDS
- Figure 5: Median survival for each WHO MDS subtype
- Figure 6: Cumulative risk of progressing to AML after 2 years for each
MDS WHO subtype
- Figure 7: International Prognostic Scoring System (IPSS) for MDS
- Figure 8: Male and female incidence of MDS in the US over and under 65
years of age as a percentage of the total US patient population, 2007
- Figure 9: MDS incidence in the seven major markets, 2007 and 2016
- Figure 10: Incidence of MDS subtypes in the seven major markets, 2007
- Figure 11: Factors complicating the management of MDS
- Figure 12: Summary of supportive care, low-intensity and
high-intensity therapies available for the management of MDS
- Figure 13: Summary of current trends in the management of lower-risk
MDS
- Figure 14: Summary of current trends in the management of higher-risk
MDS
- Figure 15: NCCN treatment guidelines for lower-risk MDS patients (IPSS
Low/Intermediate-1)
- Figure 16: The implications of long-term transfusion dependence
- Figure 17: Thrombocytopenia in MDS
- Figure 18: Phase II study of Revlimid in MDS patients with del(5q)
- Figure 19: Phase II study of Revlimid in MDS patients without del(5q)
- Figure 20: Phase II study of Revlimid in Intermediate-2/High-risk MDS
with del(5q): Interim results
- Figure 21: Phase III study of Lymphoglobuline with cyclosporine versus
best supportive care in Low/Intermediate-1 MDS
- Figure 22: NCCN treatment guidelines for IPSS Intermediate-2/High-risk
MDS patients
- Figure 23: Phase III study of Vidaza versus supportive care in MDS
patients
- Figure 24: Phase III study of Vidaza versus conventional care regimens
in higher-risk MDS
- Figure 25: Phase III study of Dacogen versus supportive care in
patients with MDS
- Figure 26: US sales of Vidaza and Dacogen, Q1 2006-Q3 2007
- Figure 27: Summary of unmet needs in the treatment of MDS
- Figure 28: Clinical development pipeline for MDS, 2007
- Figure 29: Proteins undergoing farnesylation by farnesyltransferase
- Figure 30: Phase I/II study of Sarasar in advanced MDS and CMML
- Figure 31: Phase I/II study of Romiplostim in Low-risk/Intermediate-1
MDS
- Figure 32: Proteins undergoing farnesylation by farnesyltransferase
- Figure 33: Phase II study of Zarnestra in Intermediate and High-risk
MDS
- Figure 34: Phase II study of Cloretazine in AML and high-risk MDS
- Figure 35: Phase II studies of intravenous and oral Clolar in
higher-risk MDS
- Figure 36: Phase I/II study of intravenous Telintra (liposomes for
injection) in MDS
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