title: Measuring symptoms as a critical component of drug development and evaluation in hematological diseases.
abstract: With the rapid development of new therapies for patients with hematological malignancies, there is an increasing need for patient report of symptom status during all phases of drug testing. The patient's perspective on new treatments reflects treatment tolerability as well as symptom benefit, and may assist patients and clinicians in choosing treatments. Inclusion of patient-reported outcomes, more common in solid-tumor than hematological trials, provides early information about symptoms to guide decisions about appropriate dosing and supportive care needs. We provide a historical overview of the use of patient-reported outcomes and symptom assessment in solid-tumor and hematological drug development, and offer recommendations about methodological issues in the monitoring of symptoms in the drug development process in hematological clinical trials.
authors.affiliation: Department of Symptom Research, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Unit 1450, Houston, TX 77030, USA.
authors.forename: Loretta A
authors.initials: LA
authors.lastname: Williams
authors.affiliation: Department of Symptom Research, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Unit 1450, Houston, TX 77030, USA.
authors.forename: Emre
authors.initials: E
authors.lastname: Yucel
authors.affiliation: Department of Leukemia, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Unit 428, Houston, TX 77030, USA.
authors.forename: Jorge E
authors.initials: JE
authors.lastname: Cortes
authors.affiliation: Department of Symptom Research, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Unit 1450, Houston, TX 77030, USA.
authors.forename: Charles S
authors.initials: CS
authors.lastname: Cleeland
keywords: European Medicines Agency; US FDA; cancer clinical trials; drug development; patient-reported outcomes; recommendations; symptom assessment; symptom benefit; symptom burden