Considerations for optimal HCT trial design for IBMFS
| • Prioritize prospective, multicenter trials |
| • Articulate diagnostic categorization and hematologic disease status based on molecular features, in eligibility/stratification criteria |
| • Assess adherence to eligibility criteria across centers and ensure timely revision thereof to reflect real-world experience (ie, minimize “picking and choosing”) |
| • Include broad age ranges |
| • Account for disease-specific comorbidities |
| • Develop strata encompassing MDS/AML, including de novo presentations |
| • Consider accessibility of diagnostics (eg, genetic and functional testing) and interventions (eg, graft type/manipulation) at different centers/different parts of the world |
| • Develop trials in collaboration with patient advocacy groups |
| • Include quality-of-life and long-term follow-up measures |
| • Prioritize prospective, multicenter trials |
| • Articulate diagnostic categorization and hematologic disease status based on molecular features, in eligibility/stratification criteria |
| • Assess adherence to eligibility criteria across centers and ensure timely revision thereof to reflect real-world experience (ie, minimize “picking and choosing”) |
| • Include broad age ranges |
| • Account for disease-specific comorbidities |
| • Develop strata encompassing MDS/AML, including de novo presentations |
| • Consider accessibility of diagnostics (eg, genetic and functional testing) and interventions (eg, graft type/manipulation) at different centers/different parts of the world |
| • Develop trials in collaboration with patient advocacy groups |
| • Include quality-of-life and long-term follow-up measures |