Table 3.

Explicit definitions of cure

Definition
“…[thalassemia-free] survival curves of these patients reached a plateau around 3 year after BMT and remained unchanged there-after…”45
a) “…capable of producing and maintaining a normal hemoglobin level in the recipient.”48 b) “functional graft”48 c) “developed PMC [persistent mixed chimerism]”48
“…relief from SCD and recovery after the transplant.”7
“…replaces the defective product with normal cells…”49
“…no subsequent disease-related expenditures.”47
“…‘fully effective’ cure as one that completely suppresses disease-related complications and costs and restores life expectancy and health-related quality of life (HRQoL) to that of a comparable individual unaffected by the disease.”30
“…100 percent probability of cure and 0 percent short term mortality (survival-cure).”32