Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome With Low Red Blood Cell Transfusion Burden

RBC TI was defined as the absence of any IV RBC transfusion (packed cell or whole blood) during any consecutive 56 days during the treatment period. Data presented is for number of participants with RBC TI ≥56 consecutive days since first dose in the first 28 weeks of treatment.

Time frame: 28 weeks

Population: FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment.

The RBC TI was defined as the absence of any intravenous (IV) RBC transfusion (packed cell or whole blood) during any consecutive 56 days during the treatment period. Data presented is for number of participants with RBC TI ≥8 weeks (≥56 consecutive days) since first dose in the first 28 weeks of treatment.

Time frame: 28 weeks

Population: The full analysis set (FAS) included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment hemoglobin (Hb) assessment.

Number of pRBC transfusions at baseline was defined as pRBC transfusions requirement during 8-week period prior to the start of first study medication.

Time frame: Baseline, Week 28

Population: FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment.

The PEW of TI periods over the first 28 weeks was added up to a cumulative number of weeks. For a participant with at least 1 TI response period over the first 28 weeks, the last TI response period was ended with the date of a subsequent RBC transfusion, visit date at Week 28, date of the end of study or treatment discontinuation due to AE/SAE or death, whichever came earlier. For a participant with no TI response period over the first 28 weeks, the cumulative number of PEW was set to zero.

Time frame: 28 weeks

Population: FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment.

The EQ-5D questionnaire is designed for self-completion by participants. The EQ-5D-5L descriptive system comprises the following 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problem, moderate problems, severe problems, and unable to/extreme problems. The questionnaire also included a visual analogue scale, where the participant was asked to rate current health status on a scale of 0-100, with 0 being the worst imaginable health state and 100 being the best imaginable health.

Time frame: Baseline, Week 9

Population: FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

The PROMIS physical function item measures self-reported, current capability to carry out activities that require physical actions, ranging from self-care (activities of daily living) to more complex activities that require a combination of skills, often within a social context. The PF 10-item short form which contains 10 questions was used in this study, and each item was scored on a 5-point rating scale (1 \[unable to do\] to 5 \[without any difficulty\]), with higher scores indicating better functioning. Total raw score was the sum of the response to each question, with the lowest possible raw score 10 (poor physical function) and the highest possible raw score 50 (better physical function). Raw scores converted to T-scores (as detailed in the T-score conversion table for PROMIS-SF v2.0 Physical Function 10b) with a mean of 50 and a standard deviation (SD) of 10. T-scores ranged from minimum 13.8 to maximum 61.3 possible scores with higher scores indicating better physical functioning.

Time frame: Baseline, Week 9

Population: FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Fatigue was measured using the 13-item fatigue scale of the Functional Assessment of Chronic Illness Therapy (FACIT) Measurement System, each item was scored on a 5-point rating scale ranging from 1 not at all to 5 very much, with lower scores indicating better functioning. Total raw score was the sum of the response to each question, with the lowest possible raw score 13 (lowest level of fatigue) and the highest possible raw score 65 (highest level of fatigue), with lower scores indicating better functioning. Raw scores converted to T-scores (as detailed in the T-score conversion table for PROMIS-SF v1.0 Fatigue 13a) with a mean of 50 and a SD of 10. T-scores ranged from minimum 30.3 to maximum 83.5 possible scores with lower scores indicating better functioning.

Time frame: Baseline, Week 9

Population: FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Baseline number of transfusions (pRBC/8-weeks) = total number of packs of rRBCs within 16 weeks prior to first dose/2. A pRBC transfusion reduction responder was defined as a participant who achieved ≥50% reduction in number of pRBC transfusions over 8 weeks compared to their baseline for any 8 week period in the duration begining with the first dose date (Day 1) and ending with the end of study or treatment discontinuation due to adverse event (AE)/serious adverse event (SAE) or death, whichever came earlier.

Time frame: Baseline up to Week 8

Population: FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment.

≥20 consecutive weeks TI was defined as the absence of any IV RBC transfusion (packed cell or whole blood) during any consecutive 140 days anytime during the study (up to 56 weeks). TI was estimated between the first dose date (Day 1) and the end of study (Week 56) or treatment discontinuation due to AE/SAE or death, whichever came earlier.

Time frame: Baseline up to Week 56

Population: FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment.

RBC TI was defined as the absence of any IV RBC transfusion (packed cell or whole blood) during any consecutive 56 days anytime during the study (up to Week 56).

Time frame: Baseline up to Week 56

Population: FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment.

RBC TI was defined as the absence of any IV RBC transfusion (packed cell or whole blood) during any consecutive 56 days during the treatment period. Data presented is for number of participants with RBC TI ≥56 consecutive days since first dose in the 52 weeks of treatment.

Time frame: 52 weeks

Population: FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment.

Number of pRBC transfusions at baseline was defined as pRBC transfusions requirement during 8-week period prior to the start of first study medication. Responders were defined as participants with at least a 50% reduction in the number of pRBC transfusions over any 8-week (56 consecutive days) period during the study as compared with the baseline.

Time frame: Baseline up to Week 8

Population: FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment.