The impact of a recent relapse on patient-reported outcomes in subjects with multiple sclerosis

This overview draws out the main conclusions from the 4 workshops focused on incorporating the patient perspective into outcome assessment at the 10th Outcome Measures in Rheumatology (OMERACT 10) conference. They raised methodological issues about the choice of outcome domains to include in clinical trials, the development or choice of instruments to measure these domains, and the way these instruments might capture the impact of a disease and its treatment. The need to develop a more rigorous conceptual model of quantifying the way conditions affect health, and the need to ensure patients are directly involved in the decisions about domains and instruments, emerged clearly. The OMERACT participants voted to develop guidelines for domain and instrument selection, and conceptual and experimental work will be brought forward to revise and upgrade the OMERACT Filter.

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Patient reported outcomes in patients with desmoid type fibromatosis.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.15_suppl.11574 ◽

2021 ◽

Vol 39 (15_suppl) ◽

pp. 11574-11574

Author(s):

VIKAS GARG ◽

Sameer Rastogi ◽

Adarsh Barwad ◽

Rambha Panday ◽

Sandeep Kumar Bhoriwal ◽

...

Keyword(s):

Patient Reported Outcomes ◽

Symptom Burden ◽

Benign Neoplasm ◽

Anxiety And Depression ◽

Appendicular Skeleton ◽

Healthy Controls ◽

Symptom Scale ◽

Cross Sectional ◽

Patient Reported ◽

The Impact

11574 Background: Desmoid type fibromatosis (DTF) is a rare benign neoplasm with infiltrative growth and high local recurrences. Due to long disease course, unpredictable growth pattern, and low mortality, using only survival outcomes may be inappropriate. In this study we assessed the impact of DTF on health related quality of life (HRQoL). Methods: This was a cross-sectional study done in patients with DTF. The study participants were asked to fill the EORTC QLQ-C30, GAD-7 and PHQ- 9 q uestionnaires to assess HRQoL, anxiety and depression . Outcomes were also compared with healthy controls. Results: 204 subjects (102 DTF patients and 102 healthy controls) were recruited. Study parameters have been summarized in Table. Appendicular skeleton (limbs + girdle) was most commonly involved in 59 % patients and abdominal wall or mesentery was involved in 22.5 %. Patients have received median of 2 lines of therapy. 54 % patients were currently on sorafenib and 41 % were under active surveillance. Mean global health status in DTF patient 65.58 ± 22.64, was significantly lower than healthy controls. Similarly, DTF patients scored low on all functional scales except cognitive functioning. Symptom scale showed significantly higher symptom burden of fatigue, pain, insomnia and financial difficulties. Anxiety & depression was observed in 39.22 % and 50 % of DTF patients respectively. DTF patients had higher rates of mild, moderate and severe anxiety and depression compared to healthy controls. No difference was observed based on site of disease. Conclusions: DTF patients have significant symptom burden, poor functioning, and heightened anxiety and depression. Patient reported outcomes should be routinely used to assess treatment efficacy in DTF patients.[Table: see text]

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PATIENT-REPORTED OUTCOMES AND DISABILITY IN MULTIPLE SCLEROSIS

Journal of Neurology Neurosurgery & Psychiatry ◽

10.1136/jnnp-2015-312379.127 ◽

2015 ◽

Vol 86 (11) ◽

pp. e4.32-e4

Author(s):

Neil Scolding ◽

Hongwei Wang ◽

Yan Liu ◽

Lawrence Steinman

Keyword(s):

Multiple Sclerosis ◽

Patient Reported Outcomes ◽

Short Form ◽

Minimum Important Difference ◽

Point Change ◽

Clinical Difference ◽

Sf 36 ◽

Patient Reported ◽

Edss Score ◽

Multiple Sclerosis Patients

In the 2-year, phase 3 CARE-MS II study (NCT00548405), alemtuzumab demonstrated superior clinical and patient-reported outcomes (PROs) over subcutaneous interferon beta-1a in relapsing-remitting multiple sclerosis patients who had inadequate efficacy response to prior therapy. To further evaluate the relationship between PROs and disability, Short-Form 36-Item (SF-36) survey physical component summary (PCS) and mental component summary (MCS), and Functional Assessment of Multiple Sclerosis (FAMS) scores were analysed against Expanded Disability Status Scale (EDSS) outcomes, adjusted for baseline characteristics and randomisation arm. A 1.0-point difference in baseline EDSS score was associated with 2.0-point PCS, 0.8-point MCS, and 4.0-point FAMS worsening over 12 months (all P<0.001). A 1.0-point annualised EDSS score worsening corresponded to a 2.2-point PCS, 1.6-point MCS, and 6.0-point FAMS worsening (all P<0.001). For baseline EDSS score <4.0, 1.0-point annualised worsening was associated with 7.2-point FAMS and 2.0-point MCS worsening (both P<0.001). For baseline EDSS score ≥4.0, 1.0-point worsening corresponded to worsening on FAMS (2.4 points; P=0.04), but not MCS (P=0.82). Given that a half-point EDSS change is considered the minimum reliably measurable clinical difference, a 1.0-point change in SF-36 PCS and MCS or 3.0-point change in FAMS may represent a minimum important difference in PRO for multiple sclerosis patients.

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