New approach to AE reporting needed, group says


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Doctor and patient

A group of experts has called for improvements in reporting adverse events (AEs) that occur in patients with hematologic malignancies.

The group highlighted deficiencies in capturing chronic, cumulative, and late AEs; collecting patient-reported outcomes (PROs); reporting AEs associated with hematopoietic stem cell transplant (HSCT); assessing long-term toxicity in survivors; reporting AEs to regulatory agencies; and tracking AEs that occur in routine clinical practice.

The experts discussed these problems and made recommendations for fixing them in The Lancet Haematology.

“The success in outcomes and survival in many hematological malignancies is historically unparalleled and fueled by scientific discovery and implementation,” said author Gita Thanarajasingam, MD, of the Mayo Clinic in Rochester, Minnesota.

“Patients are now living with the challenge of managing not just their hematological malignancy but also managing chronic therapy for their illness, with new types of acute, chronic, cumulative, and late toxicities. Measures to address the broad facets of toxicity assessment must be prioritized and further developed to ultimately enhance accurate, comprehensive, patient-centered toxicity reporting that will meaningfully inform the care of patients with blood cancers.”

Dr Thanarajasingam and a group of clinicians, investigators, regulators, biostatisticians, and patient advocates analyzed the evidence on AEs and proposed recommendations to policy makers, researchers, industry, and regulators.

First, the group noted that chronic, delayed, and cumulative AEs may go unreported in patients with hematologic malignancies. Therefore, longitudinal methods for AE analysis are needed, and phase 1 trials should have longer periods for evaluating dose-limiting toxicity.

The experts also said PROs are often overlooked, but it should be standard to assess PROs in clinical trials of patients with hematologic malignancies.

Another of the group’s concerns is the “cumbersome” reporting of AEs associated with HSCT acting as a barrier to clinical trials. The experts recommended using registry data to develop a consensus on expected AEs after HSCT.

The group also highlighted deficiencies in the “description and management” of cumulative and late toxicities in survivors of hematologic malignancies. Potential solutions to this problem include developing infrastructure to collect data for adult survivors and standardizing the use and content of survivorship care plans.

The experts said “meaningful” AEs are often underreported to regulatory agencies, so better systems are needed for collecting and analyzing AE data, and the electronic submission of AEs should be simplified.

Finally, the group said AEs occurring in routine clinical practice are difficult to capture and analyze on a large scale. This suggests a need to optimize the systematic, objective collection of toxicity data at multiple points in real-world databases, according to the experts.

Additional details and recommendations are available in the full report.

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