Methodology for clinical trials involving patients with cancer who have febrile neutropenia: updated guidelines of the Immunocompromised Host Society/Multinational Association for Supportive Care in Cancer, with emphasis on outpatient studies.
Two multinational organizations, the Immunocompromised Host Society and the Multinational Association for Supportive Care in Cancer, have produced for investigators and regulatory bodies a set of guidelines on methodology for clinical trials involving patients with febrile neutropenia. The guidelines suggest that response (i.e., success of initial empirical antibiotic therapy without any modification) be determined at 72 h and again on day 5, and the reasons for modification should be stated. Blinding and stratification are to be encouraged, as should statistical consideration of trials specifically designed for showing equivalence. Patients enrolled in outpatient studies should be selected by use of a validated risk model, and patients should be carefully monitored after discharge from the hospital. Response and safety parameters should be recorded along with readmission rates. If studies use these guidelines, comparisons between studies will be simpler and will lead to further improvements in patient therapy.
Feld
Ronald
R
Department of Hematology and Oncology, Princess Margaret Hospital, Toronto, Ontario, Canada M5G 2M9. ronald.feld@uhn.on.ca
Paesmans
Marianne
M
Freifeld
Alison G
AG
Klastersky
Jean
J
Pizzo
Philip A
PA
Rolston
Kenneth V I
KV
Rubenstein
Edward
E
Talcott
James A
JA
Walsh
Thomas J
TJ
D000900:Anti-Bacterial Agents / Q000627:therapeutic use; D002986:Clinical Trials as Topic / Q000379:methods*; D004311:Double-Blind Method; D005334:Fever / Q000209:etiology; D006801:Humans; D016867:Immunocompromised Host; D009369:Neoplasms / Q000503:physiopathology*; D009503:Neutropenia / Q000188:drug therapy / Q000209:etiology*; D010045:Outpatients; D018579:Patient Selection
D000900:Anti-Bacterial Agents