Table 1.

Subgroups in the EMA/PDCO AML standard PIP

• Patients with newly-diagnosed high-risk AML: need for a more efficacious treatment as part of a first-line induction regimen, in particular when there is a good rationale for use during first-line treatment, such as the individual disease biology (eg, FLT3 mutations with high allelic ratio etc) or the potential for reduction of toxicity.

• Patients with AML that is resistant to first or to second line induction treatment: need for an efficacious treatment as part of a re-induction regimen.

• Patients at the time of diagnosis of relapse after HSCT/second or subsequent relapse: need for an efficacious treatment that is not overly toxic in this subset of patients who likely had high cumulative previous treatment exposure, likely including at least one prior transplant procedure.

• Patients with secondary AML: need for an efficacious treatment.

• Patients at the time of diagnosis of early first relapse: need for a more efficacious treatment as part of a treatment regimen.

• Patients at the time of diagnosis of first relapse (other than early): need for a more efficacious treatment as part of a treatment regimen.

• Patients with APL: need for safer treatment to be used during induction.

• Patients with AML in Down syndrome: Needs may exist, specifically for non-cytotoxic or “targeted” medicines to reduce treatment toxicity. Needs may be less in patients younger than 1 year of age and in those with FAB M6 or M7, compared to other patients with AML in Down syndrome.

• Congenital AML, extramedullary AML.

Notes: The content of this table contains the wording of the AML pediatric subgroups from the AML standard PIP. Underlinings, explanations in brackets, and quotation marks are exact copies of the AML standard PIP.

Abbreviations: EMA, European Medicines Agency; PDCO, pediatric committee; AML, acute myeloid leukemia; PIP, pediatric investigation plan; HSCT, hematopoietic stem cell transplantation; APL, acute promyelocytic leukemia; FAB, French–American–British.