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Patient-Reported Outcome Measures Pick Up Patient Experiences in Patients With TTP


Patients with thrombotic thrombocytopenic purpura (TTP) benefited from patient-reported outcome measures to report their quality of life.

Quality of life measures could be assessed using patient-reported outcome measures (PROMs) in patients diagnosed with thrombotic thrombocytopenic purpura (TTP), according to a review published in the Journal of Clinical Medicine.

TTP is a rare blood disorder that can affect health-related quality of life (HRQoL). PROMs have been used to measure HRQoL, as they can assess 1 or multiple outcomes from a patient perspective and come directly from the patient themselves. It is not known if PROMs are being used in clinical settings or if they can be used to capture HRQoL effects that are specific to TTP. This review aimed to “define the landscape of PROMs at present and their validity evidence in studies of patients with TTP.”

The researchers searched the databases of MEDLINE (PubMed), Embase (Elsevier), Scopus (Elsevier), and CINAHL (EBSCO) from their respective inceptions until June 10, 2022. An updated search was done on October 10, 2022. Studies were included if the participants had a clinical diagnosis of TTP and if they reported on the use of PROMs. If the study included patients with only hereditary TTP, PROMs were not evaluated, or PRO results were not reported, the study was excluded. All studies were assessed for quality using the Joanna Briggs Institute Clinical Appraisal Tools checklist.

There were 14 studies that covered 16 PROMs in 970 patients that were included in the review with 5 cross-sectional studies, 8 cohort studies, and 1 clinical trial. There were no studies that reported on PROMs that were specific to TTP and none assessed the effect of integrating PROMs into clinical practice for TTP. Only 1 study assessed PROMs in pediatric patients but the results were not reported.

HRQoL, depression, and anxiety were the 3 most common domains that were assessed, with 8 studies using 3 different PROMS to assess overall HRQoL. Depression was assessed using 6 different PROMs in 8 studies. A total of 3 different PROMs in 3 studies were used to assess anxiety in patients.

The PROMs found that patients’ HRQoL were affected after an acute episode of TTP. Patients with TTP were also found to have worse scores in the HRQoL compared with the general population of the United States and Italy when using PROMs. Patients with TTP also had similar or worse scores when compared with chronic conditions such as anemia, cnacer, and depression. A higher prevalence of depression and anxiety was found in patients with TTP compared with controls. Post-traumatic stress disorder screens were also prevalent in patients with TTP.

Time between an episode of TTP and administering a PROM was only recorded in 2 studies; the researchers found the median time in these 2 studies was 6.3 and 6.6 years from TTP episode to the administering of a PROM. Depression and anxiety scores were not found to be associated with the number of TTP episodes, neurological symptoms, ADAMTS13 activity during remission, and abnormal MRIs.

There were some limitations to this study. PROMs may have been distributed to the same population multiple times, which could lead to an overlap of patients in studies. Patients with other types of thrombotic microangiopathy could have been included in the study due to ADAMTS13 activity not being used as inclusion criterion. Some concepts could into be assessed due to the qualitative studies being used in the post-hoc analysis. Comparisons across studies could not be done due to the heterogeneity in domains assessed.

The researchers concluded that more studies should be done to assess how PROMs can work for patients with TTP and whether existing PROMs can be modified for the use in patients with TTP.


Soares Ferreira Junior A, Pinheiro Maux Lessa M, Kaplan S, et al. Patient-reported outcome measures in patients with thrombotic thrombocytopenic purpura: a systematic review of the literature. J Clin Med. 2023;12:5155. doi:10.3390/jcm12155155

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