Trial Outcomes & Findings for Aralast alpha1-proteinase Inhibitor Surveillance Study (NCT NCT00313144)

NCT ID: NCT00313144

Last Updated: 2021-05-26

Results Overview

Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on: Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000.

• Recruitment status: TERMINATED
• Study phase: PHASE4
• Target enrollment: 127 participants
• Primary outcome timeframe: Screening to ≤ 6 Months
• Results posted on: 2021-05-26

Participant Flow

This was a surveillance study conducted by Coram, Inc., in locations throughout the United States, and supervised by Baxter. Enrollment began in June 2006 and the study was terminated early, in December 2008, due to Aralast being phased out of the market.

127 participants enrolled in the study, of these 126 participated in the health related quality of life (HRQoL) and healthcare resource utilization (HCRU) portion, 61 consented to the blood draw portion, and 66 only consented to the Quality of Life portion

Participant milestones

Measure	Overall Study Participants were treated with ARALAST according to dose and frequency of infusions recommended by their physician.
Overall Study STARTED	127
Overall Study COMPLETED	17
Overall Study NOT COMPLETED	110

Reasons for withdrawal

Measure	Overall Study Participants were treated with ARALAST according to dose and frequency of infusions recommended by their physician.
Overall Study Discontinue Aralast ≥4 consecutive doses	12
Overall Study Death	7
Overall Study Lost to Follow-up	5
Overall Study Withdrawal by Subject	4
Overall Study Terminated by sponsor/ homecare provider	4
Overall Study Physician Decision	4
Overall Study Adverse event - transient	5
Overall Study Liver transplant	1
Overall Study Insurance (homecare provider) change	1
Overall Study Change to different product	1
Overall Study Due to termination of the study	66

Baseline Characteristics

Aralast alpha1-proteinase Inhibitor Surveillance Study

Baseline characteristics by cohort

Measure	Overall Study n=127 Participants
Age, Continuous	52.3 years n=5 Participants
Sex: Female, Male Female	68 Participants n=5 Participants
Sex: Female, Male Male	59 Participants n=5 Participants
Region of Enrollment United States	127 Participants n=5 Participants
Health Related Quality of Life (HRQoL): Baseline Assessment SF-36 Health Domain Scores (n=126) Physical Functioning (PF)	31.612 Scores on a scale STANDARD_DEVIATION 11.678 • n=5 Participants
Health Related Quality of Life (HRQoL): Baseline Assessment SF-36 Health Domain Scores (n=126) Role Limitation Due to Physical Health (RP)	37.281 Scores on a scale STANDARD_DEVIATION 11.871 • n=5 Participants
Health Related Quality of Life (HRQoL): Baseline Assessment SF-36 Health Domain Scores (n=126) Bodily Pain (BP)	46.368 Scores on a scale STANDARD_DEVIATION 11.116 • n=5 Participants
Health Related Quality of Life (HRQoL): Baseline Assessment SF-36 Health Domain Scores (n=126) General Health (GH)	37.351 Scores on a scale STANDARD_DEVIATION 10.120 • n=5 Participants
Health Related Quality of Life (HRQoL): Baseline Assessment SF-36 Health Domain Scores (n=126) Vitality (VT)	42.279 Scores on a scale STANDARD_DEVIATION 11.209 • n=5 Participants
Health Related Quality of Life (HRQoL): Baseline Assessment SF-36 Health Domain Scores (n=126) Social Functioning (SF)	41.699 Scores on a scale STANDARD_DEVIATION 14.069 • n=5 Participants
Health Related Quality of Life (HRQoL): Baseline Assessment SF-36 Health Domain Scores (n=126) Role Limitation Due to Emotional Problems (RE)	48.474 Scores on a scale STANDARD_DEVIATION 10.687 • n=5 Participants
Health Related Quality of Life (HRQoL): Baseline Assessment SF-36 Health Domain Scores (n=126) Mental Health (MH)	47.326 Scores on a scale STANDARD_DEVIATION 11.358 • n=5 Participants
Health Related Quality of Life (HRQoL): Baseline Assessment SF-36 Health Domain Scores (n=126) Physical Component Score (PCS)	34.150 Scores on a scale STANDARD_DEVIATION 11.263 • n=5 Participants
Health Related Quality of Life (HRQoL): Baseline Assessment SF-36 Health Domain Scores (n=126) Mental Component Score (MCS)	50.340 Scores on a scale STANDARD_DEVIATION 11.897 • n=5 Participants

PRIMARY outcome

Timeframe: Screening to ≤ 6 Months

Population: Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL

Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on: Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000.

Outcome measures

Measure	Baseline n=106 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months	>12 Months to ≤18 Months	>18 Months to ≤24 Months
HRQoL 'Physical Functioning (PF)' From Baseline to ≤6 Months	31.958 Scores on a scale Standard Deviation 11.291	33.129 Scores on a scale Standard Deviation 11.527	—	—	—

PRIMARY outcome

Timeframe: Screening to ≤ 6 Months

Population: Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL

Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000.

Outcome measures

Measure	Baseline n=106 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months	>12 Months to ≤18 Months	>18 Months to ≤24 Months
HRQoL 'Role Limitation Due to Physical Health (RP)' From Baseline to ≤6 Months	37.631 Scores on a scale Standard Deviation 11.819	38.139 Scores on a scale Standard Deviation 11.338	—	—	—

PRIMARY outcome

Timeframe: Screening to ≤ 6 Months

Population: Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL

Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000.

Outcome measures

Measure	Baseline n=106 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months	>12 Months to ≤18 Months	>18 Months to ≤24 Months
HRQoL 'Bodily Pain (BP)' From Baseline to ≤6 Months	46.474 Scores on a scale Standard Deviation 10.895	48.950 Scores on a scale Standard Deviation 11.579	—	—	—

PRIMARY outcome

Timeframe: Screening to ≤ 6 Months

Population: Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL

Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000.

Outcome measures

Measure	Baseline n=106 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months	>12 Months to ≤18 Months	>18 Months to ≤24 Months
HRQoL 'General Health (GH)' From Baseline to ≤6 Months	37.365 Scores on a scale Standard Deviation 10.038	38.737 Scores on a scale Standard Deviation 10.490	—	—	—

PRIMARY outcome

Timeframe: Screening to ≤ 6 Months

Population: Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL

Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000.

Outcome measures

Measure	Baseline n=106 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months	>12 Months to ≤18 Months	>18 Months to ≤24 Months
HRQoL 'Vitality (VT)' From Baseline to ≤6 Months	42.578 Scores on a scale Standard Deviation 11.164	45.081 Scores on a scale Standard Deviation 10.916	—	—	—

PRIMARY outcome

Timeframe: Screening to ≤ 6 Months

Population: Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL

Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000.

Outcome measures

Measure	Baseline n=106 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months	>12 Months to ≤18 Months	>18 Months to ≤24 Months
HRQoL 'Social Functioning (SF)' From Baseline to ≤6 Months	42.340 Scores on a scale Standard Deviation 14.060	45.581 Scores on a scale Standard Deviation 12.780	—	—	—

PRIMARY outcome

Timeframe: Screening to ≤ 6 Months

Population: Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL

Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000.

Outcome measures

Measure	Baseline n=106 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months	>12 Months to ≤18 Months	>18 Months to ≤24 Months
HRQoL 'Role Limitation Due to Emotional Problems (RE)' From Baseline to ≤6 Months	48.361 Scores on a scale Standard Deviation 10.972	47.921 Scores on a scale Standard Deviation 10.855	—	—	—

PRIMARY outcome

Timeframe: Screening to ≤ 6 Months

Population: Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL

Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The data transformation process was based on Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000.

Outcome measures

Measure	Baseline n=106 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months	>12 Months to ≤18 Months	>18 Months to ≤24 Months
HRQoL 'Mental Health (MH)' From Baseline to ≤6 Months	47.457 Scores on a scale Standard Deviation 10.711	50.007 Scores on a scale Standard Deviation 9.480	—	—	—

PRIMARY outcome

Timeframe: Screening to ≤ 6 Months

Population: Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL

SF-36 scores for baseline (screening) versus the period from baseline to ≤6 Months. The PCS is a summary scale of the dimensions physical functioning, role physical, bodily pain, and general health. The component score is normalized to a standard population. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores.

Outcome measures

Measure	Baseline n=106 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months	>12 Months to ≤18 Months	>18 Months to ≤24 Months
HRQoL 'Physical Component Score (PCS)' From Baseline to ≤6 Months	34.453 Scores on a scale Standard Deviation 10.957	35.826 Scores on a scale Standard Deviation 10.881	—	—	—

PRIMARY outcome

Timeframe: Screening to ≤ 6 Months

Population: Participants with baseline and participating during the period from baseline to ≤6 months data for HRQoL

SF-36 scores for baseline (screening) versus the period from baseline to ≤6 Months. The MCS is a summary scale of the dimensions vitality, social functioning, role emotional, and mental health Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores.

Outcome measures

Measure	Baseline n=106 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months	>12 Months to ≤18 Months	>18 Months to ≤24 Months
HRQoL 'Mental Component Score (MCS)' From Baseline to ≤6 Months	50.464 Scores on a scale Standard Deviation 11.713	52.378 Scores on a scale Standard Deviation 10.639	—	—	—

PRIMARY outcome

Timeframe: Baseline to 12 months

Population: Participants with baseline and participating during the period from baseline to ≤12 months data for HRQoL

SF-36 Scores- baseline thru 12 months, where data was available. Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The Data transformation process was based on: Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000.

Outcome measures

Measure	Baseline n=65 Participants	Baseline to ≤6 Months n=65 Participants	>6 Months to ≤12 Months n=65 Participants	>12 Months to ≤18 Months	>18 Months to ≤24 Months
HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months Bodily Pain (BP)	47.203 Scores on a scale Standard Deviation 11.375	50.227 Scores on a scale Standard Deviation 11.267	47.788 Scores on a scale Standard Deviation 13.139	—	—
HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months General Health (GH)	38.853 Scores on a scale Standard Deviation 10.197	39.990 Scores on a scale Standard Deviation 10.013	40.026 Scores on a scale Standard Deviation 11.720	—	—
HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months Physical Functioning (PF)	34.822 Scores on a scale Standard Deviation 11.173	35.632 Scores on a scale Standard Deviation 11.251	34.143 Scores on a scale Standard Deviation 11.914	—	—
HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months Role Limitation Due to Physical Health (RP)	40.539 Scores on a scale Standard Deviation 11.627	39.484 Scores on a scale Standard Deviation 11.615	38.542 Scores on a scale Standard Deviation 13.061	—	—
HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months Vitality (VT)	44.646 Scores on a scale Standard Deviation 11.546	45.703 Scores on a scale Standard Deviation 11.516	44.742 Scores on a scale Standard Deviation 11.058	—	—
HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months Social Functioning (SF)	44.683 Scores on a scale Standard Deviation 13.864	47.200 Scores on a scale Standard Deviation 11.799	43.927 Scores on a scale Standard Deviation 14.384	—	—
HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months Role Limitation Due to Emotional Problems (RE)	48.403 Scores on a scale Standard Deviation 11.214	48.583 Scores on a scale Standard Deviation 10.590	48.942 Scores on a scale Standard Deviation 12.011	—	—
HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months Mental Health (MH)	47.365 Scores on a scale Standard Deviation 11.040	50.311 Scores on a scale Standard Deviation 8.962	49.488 Scores on a scale Standard Deviation 11.705	—	—
HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months Physical Component Score (PCS)	37.346 Scores on a scale Standard Deviation 10.906	37.890 Scores on a scale Standard Deviation 10.499	36.272 Scores on a scale Standard Deviation 11.238	—	—
HRQoL For: PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS: Baseline, Baseline to ≤6 Months, and >6 Months to ≤12 Months Mental Component Score (MCS)	50.442 Scores on a scale Standard Deviation 11.896	52.508 Scores on a scale Standard Deviation 10.354	51.854 Scores on a scale Standard Deviation 12.088	—	—

PRIMARY outcome

Timeframe: Baseline to 12 months

Population: Participants with baseline and participating during the period from baseline to ≤18 months data for HRQoL

SF-36 Scores- baseline thru 12 months, where data was available. Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The Data transformation process was based on: Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000.

Outcome measures

Measure	Baseline n=47 Participants	Baseline to ≤6 Months n=47 Participants	>6 Months to ≤12 Months n=47 Participants	>12 Months to ≤18 Months n=47 Participants	>18 Months to ≤24 Months
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, and >12 Months to ≤18 Months Role Limitation Due to Physical Health (RP)	40.492 Scores on a scale Standard Deviation 12.310	39.033 Scores on a scale Standard Deviation 11.686	37.887 Scores on a scale Standard Deviation 12.964	40.336 Scores on a scale Standard Deviation 12.324	—
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, and >12 Months to ≤18 Months Physical Functioning (PF)	34.197 Scores on a scale Standard Deviation 11.375	34.958 Scores on a scale Standard Deviation 11.048	33.301 Scores on a scale Standard Deviation 11.764	33.839 Scores on a scale Standard Deviation 12.546	—
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, and >12 Months to ≤18 Months Bodily Pain (BP)	47.525 Scores on a scale Standard Deviation 12.023	50.726 Scores on a scale Standard Deviation 10.942	48.990 Scores on a scale Standard Deviation 13.177	50.537 Scores on a scale Standard Deviation 11.789	—
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, and >12 Months to ≤18 Months General Health (GH)	38.684 Scores on a scale Standard Deviation 10.164	39.718 Scores on a scale Standard Deviation 9.697	39.475 Scores on a scale Standard Deviation 11.435	39.586 Scores on a scale Standard Deviation 10.848	—
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, and >12 Months to ≤18 Months Vitality (VT)	45.714 Scores on a scale Standard Deviation 11.843	46.245 Scores on a scale Standard Deviation 10.807	45.249 Scores on a scale Standard Deviation 11.002	45.648 Scores on a scale Standard Deviation 10.901	—
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, and >12 Months to ≤18 Months Social Functioning (SF)	45.825 Scores on a scale Standard Deviation 14.089	47.218 Scores on a scale Standard Deviation 11.665	44.781 Scores on a scale Standard Deviation 14.110	45.013 Scores on a scale Standard Deviation 14.127	—
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, and >12 Months to ≤18 Months Role Limitation Due to Emotional Problems (RE)	49.014 Scores on a scale Standard Deviation 11.497	48.187 Scores on a scale Standard Deviation 11.158	48.022 Scores on a scale Standard Deviation 13.331	47.360 Scores on a scale Standard Deviation 11.930	—
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, and >12 Months to ≤18 Months Mental Health (MH)	48.390 Scores on a scale Standard Deviation 11.740	50.187 Scores on a scale Standard Deviation 9.347	49.229 Scores on a scale Standard Deviation 12.562	48.569 Scores on a scale Standard Deviation 11.902	—
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, and >12 Months to ≤18 Months Physical Component Score (PCS)	36.803 Scores on a scale Standard Deviation 11.105	37.655 Scores on a scale Standard Deviation 10.609	36.171 Scores on a scale Standard Deviation 10.848	38.060 Scores on a scale Standard Deviation 11.450	—
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, and >12 Months to ≤18 Months Mental Component Score (MCS)	51.885 Scores on a scale Standard Deviation 12.215	52.575 Scores on a scale Standard Deviation 10.846	51.843 Scores on a scale Standard Deviation 13.323	50.814 Scores on a scale Standard Deviation 12.729	—

PRIMARY outcome

Timeframe: Baseline to 24 months

Population: Participants with baseline and participating during the period from baseline to ≤24 months data for HRQoL

SF-36 Scores- baseline thru 24 months, where data was available. Change in quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. The Data transformation process was based on: Ware et al. How to Score Version 2 of the SF-36® Health Survey. Lincoln, RI: Quality Metric Incorporated; 2000.

Outcome measures

Measure	Baseline n=21 Participants	Baseline to ≤6 Months n=21 Participants	>6 Months to ≤12 Months n=21 Participants	>12 Months to ≤18 Months n=21 Participants	>18 Months to ≤24 Months n=21 Participants
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months Vitality (VT)	47.036 Scores on a scale Standard Deviation 11.949	48.969 Scores on a scale Standard Deviation 10.116	49.118 Scores on a scale Standard Deviation 10.187	47.036 Scores on a scale Standard Deviation 9.797	47.631 Scores on a scale Standard Deviation 10.705
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months Social Functioning (SF)	46.980 Scores on a scale Standard Deviation 16.418	48.538 Scores on a scale Standard Deviation 12.026	44.123 Scores on a scale Standard Deviation 14.361	46.980 Scores on a scale Standard Deviation 13.756	48.019 Scores on a scale Standard Deviation 15.181
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months Physical Functioning (PF)	36.489 Scores on a scale Standard Deviation 11.174	35.988 Scores on a scale Standard Deviation 10.874	34.585 Scores on a scale Standard Deviation 12.241	35.487 Scores on a scale Standard Deviation 12.782	33.883 Scores on a scale Standard Deviation 12.836
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months Role Limitation Due to Physical Health (RP)	43.092 Scores on a scale Standard Deviation 13.267	42.509 Scores on a scale Standard Deviation 11.390	40.643 Scores on a scale Standard Deviation 13.245	42.276 Scores on a scale Standard Deviation 11.577	41.576 Scores on a scale Standard Deviation 12.841
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months Bodily Pain (BP)	52.036 Scores on a scale Standard Deviation 13.330	53.988 Scores on a scale Standard Deviation 11.695	52.559 Scores on a scale Standard Deviation 14.045	56.041 Scores on a scale Standard Deviation 10.951	51.674 Scores on a scale Standard Deviation 12.214
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months General Health (GH)	38.452 Scores on a scale Standard Deviation 11.496	40.744 Scores on a scale Standard Deviation 10.222	39.632 Scores on a scale Standard Deviation 11.720	40.245 Scores on a scale Standard Deviation 11.852	38.293 Scores on a scale Standard Deviation 11.171
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months Role Limitation Due to Emotional Problems (RE)	49.215 Scores on a scale Standard Deviation 14.077	49.400 Scores on a scale Standard Deviation 12.002	49.770 Scores on a scale Standard Deviation 12.984	49.770 Scores on a scale Standard Deviation 12.142	49.030 Scores on a scale Standard Deviation 12.348
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months Mental Health (MH)	48.801 Scores on a scale Standard Deviation 13.404	50.007 Scores on a scale Standard Deviation 10.686	50.812 Scores on a scale Standard Deviation 13.626	49.471 Scores on a scale Standard Deviation 12.581	50.276 Scores on a scale Standard Deviation 13.084
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months Physical Component Score (PCS)	39.963 Scores on a scale Standard Deviation 11.722	40.480 Scores on a scale Standard Deviation 11.113	38.287 Scores on a scale Standard Deviation 12.037	40.716 Scores on a scale Standard Deviation 10.711	37.890 Scores on a scale Standard Deviation 12.785
HRQoL for PF, RP, BP, GH, VT, SF, RE, MH, PCS, and MCS Scores: Baseline, Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months Mental Component Score (MCS)	51.510 Scores on a scale Standard Deviation 14.484	53.011 Scores on a scale Standard Deviation 12.441	53.120 Scores on a scale Standard Deviation 13.910	51.990 Scores on a scale Standard Deviation 12.726	53.389 Scores on a scale Standard Deviation 15.119

SECONDARY outcome

Timeframe: Baseline to 24 Months

Population: Participants with data available during each window period

Number of participants with indicated number of ER visits (0, 1, 2, 3, ≥4 ER visits per participant) during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months)

Outcome measures

Measure	Baseline n=106 Participants	Baseline to ≤6 Months n=65 Participants	>6 Months to ≤12 Months n=47 Participants	>12 Months to ≤18 Months n=21 Participants	>18 Months to ≤24 Months
Healthcare Resource Utilization (HCRU) 'Frequency of Emergency Room (ER) Visits' 0 ER visits	84 Participants	49 Participants	41 Participants	19 Participants	—
Healthcare Resource Utilization (HCRU) 'Frequency of Emergency Room (ER) Visits' 1 ER visits	16 Participants	14 Participants	5 Participants	2 Participants	—
Healthcare Resource Utilization (HCRU) 'Frequency of Emergency Room (ER) Visits' 2 ER visits	5 Participants	2 Participants	1 Participants	0 Participants	—
Healthcare Resource Utilization (HCRU) 'Frequency of Emergency Room (ER) Visits' 3 ER visits	1 Participants	0 Participants	0 Participants	0 Participants	—
Healthcare Resource Utilization (HCRU) 'Frequency of Emergency Room (ER) Visits' ≥4 ER visits	0 Participants	0 Participants	0 Participants	0 Participants	—

SECONDARY outcome

Timeframe: One year prior to baseline to 24 months post-baseline

Population: Participants with data available during each window period

Mean number of ER visits one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months)

Outcome measures

Measure	Baseline n=126 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months n=65 Participants	>12 Months to ≤18 Months n=47 Participants	>18 Months to ≤24 Months n=21 Participants
Healthcare Resource Utilization (HCRU) 'Mean Number of Emergency Room (ER) Visits'	0.85 ER visits per time period Standard Deviation 1.492	0.274 ER visits per time period Standard Deviation 0.594	0.28 ER visits per time period Standard Deviation 0.516	0.15 ER visits per time period Standard Deviation 0.416	0.10 ER visits per time period Standard Deviation 0.301

SECONDARY outcome

Timeframe: Baseline to 24 Months

Population: Participants with data available during each window period

Number of participants with indicated number of hospitalizations during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months)

Outcome measures

Measure	Baseline n=106 Participants	Baseline to ≤6 Months n=65 Participants	>6 Months to ≤12 Months n=47 Participants	>12 Months to ≤18 Months n=21 Participants	>18 Months to ≤24 Months
Healthcare Resource Utilization (HCRU) 'Frequency of Hospitalizations' 0 Hospitalizations	86 Participants	58 Participants	45 Participants	20 Participants	—
Healthcare Resource Utilization (HCRU) 'Frequency of Hospitalizations' 1 Hospitalizations	17 Participants	6 Participants	1 Participants	1 Participants	—
Healthcare Resource Utilization (HCRU) 'Frequency of Hospitalizations' 2 Hospitalizations	2 Participants	1 Participants	1 Participants	0 Participants	—
Healthcare Resource Utilization (HCRU) 'Frequency of Hospitalizations' 3 Hospitalizations	1 Participants	0 Participants	0 Participants	0 Participants	—
Healthcare Resource Utilization (HCRU) 'Frequency of Hospitalizations' ≥4 Hospitalizations	0 Participants	0 Participants	0 Participants	0 Participants	—

SECONDARY outcome

Timeframe: Baseline to 24 months

Population: Participants with data available during each window period

Mean LOS during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months)

Outcome measures

Measure	Baseline n=106 Participants	Baseline to ≤6 Months n=65 Participants	>6 Months to ≤12 Months n=47 Participants	>12 Months to ≤18 Months n=21 Participants	>18 Months to ≤24 Months
Healthcare Resource Utilization (HCRU) 'Mean Length of Stay (LOS) in Hospital'	0.8585 Days Standard Deviation 2.29467	0.6462 Days Standard Deviation 2.68346	0.25532 Days Standard Deviation 1.374742	1.1905 Days Standard Deviation 5.45545	—

SECONDARY outcome

Timeframe: One year prior to baseline to 24 months post-baseline

Population: Participants with data available during each window period

Number of participants taking antibiotics one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months)

Outcome measures

Measure	Baseline n=127 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months n=65 Participants	>12 Months to ≤18 Months n=47 Participants	>18 Months to ≤24 Months n=21 Participants
Healthcare Resource Utilization (HCRU) 'Number of Participants Taking Antibiotics'	90 Participants	58 Participants	37 Participants	25 Participants	8 Participants

SECONDARY outcome

Timeframe: One year prior to baseline to 24 months post-baseline

Population: Participants with data available during each window period

Number of antibiotic courses (i.e. number of antibiotic prescriptions) one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months)

Outcome measures

Measure	Baseline n=126 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months n=65 Participants	>12 Months to ≤18 Months n=47 Participants	>18 Months to ≤24 Months n=21 Participants
Healthcare Resource Utilization (HCRU) 'Number of Antibiotic Courses' All antibiotic courses (oral and I.V.)	2.3095 Antibiotic courses Standard Deviation 2.84384	0.9906 Antibiotic courses Standard Deviation 1.27612	0.9231 Antibiotic courses Standard Deviation 1.07975	0.9787 Antibiotic courses Standard Deviation 1.40634	0.7143 Antibiotic courses Standard Deviation 1.18924
Healthcare Resource Utilization (HCRU) 'Number of Antibiotic Courses' Oral antibiotic courses	1.8571 Antibiotic courses Standard Deviation 2.39571	0.8679 Antibiotic courses Standard Deviation 1.08738	0.8154 Antibiotic courses Standard Deviation 0.96651	0.8936 Antibiotic courses Standard Deviation 1.25515	0.6667 Antibiotic courses Standard Deviation 1.19722
Healthcare Resource Utilization (HCRU) 'Number of Antibiotic Courses' I.V. antibiotic courses	0.4603 Antibiotic courses Standard Deviation 1.08555	0.1038 Antibiotic courses Standard Deviation 0.36330	0.1077 Antibiotic courses Standard Deviation 0.35895	0.0851 Antibiotic courses Standard Deviation 0.28206	0.0476 Antibiotic courses Standard Deviation 0.21822

SECONDARY outcome

Timeframe: One year prior to baseline to 24 months post-baseline

Population: Participants with data available during each window period

Number of participants receiving steroid pulse courses (i.e. number of steroid prescriptions) one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months)

Outcome measures

Measure	Baseline n=127 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months n=65 Participants	>12 Months to ≤18 Months n=47 Participants	>18 Months to ≤24 Months n=21 Participants
Healthcare Resource Utilization (HCRU) 'Number of Participants Receiving Steroid Pulse Courses'	65 Participants	30 Participants	14 Participants	12 Participants	4 Participants

SECONDARY outcome

Timeframe: One year prior to baseline to 24 months post-baseline

Population: Participants with data available during each window period

Number of steroid pulse courses (i.e. number of steroid prescriptions) one year prior to baseline/screening, and during each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months)

Outcome measures

Measure	Baseline n=126 Participants	Baseline to ≤6 Months n=106 Participants	>6 Months to ≤12 Months n=65 Participants	>12 Months to ≤18 Months n=47 Participants	>18 Months to ≤24 Months n=21 Participants
Healthcare Resource Utilization (HCRU) 'Number of Steroid Pulse Courses' All steroid pulse courses (oral and I.V.)	1.4365 Steroid Pulse Courses Standard Deviation 2.51554	0.4340 Steroid Pulse Courses Standard Deviation 0.89463	0.4154 Steroid Pulse Courses Standard Deviation 1.07372	0.3404 Steroid Pulse Courses Standard Deviation 0.66844	0.2857 Steroid Pulse Courses Standard Deviation 0.71714
Healthcare Resource Utilization (HCRU) 'Number of Steroid Pulse Courses' Oral steroid pulse courses	1.0873 Steroid Pulse Courses Standard Deviation 2.13174	0.3585 Steroid Pulse Courses Standard Deviation 0.84164	0.3692 Steroid Pulse Courses Standard Deviation 1.03937	0.2766 Steroid Pulse Courses Standard Deviation 0.53981	0.2381 Steroid Pulse Courses Standard Deviation 0.70034
Healthcare Resource Utilization (HCRU) 'Number of Steroid Pulse Courses' I.V. steroid pulse courses	0.3492 Steroid Pulse Courses Standard Deviation 0.96596	0.0849 Steroid Pulse Courses Standard Deviation 0.28007	0.0462 Steroid Pulse Courses Standard Deviation 0.21145	0.0638 Steroid Pulse Courses Standard Deviation 0.24709	0.0476 Steroid Pulse Courses Standard Deviation 0.21822

SECONDARY outcome

Timeframe: Baseline to 24 months

Population: Subjects who participated in the blood draws with data available during each window period

Summary of changes in hepatic (total bilirubin, aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase) parameters from screening/baseline through each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months)

Outcome measures

Measure	Baseline n=52 Participants	Baseline to ≤6 Months n=34 Participants	>6 Months to ≤12 Months n=26 Participants	>12 Months to ≤18 Months n=15 Participants	>18 Months to ≤24 Months
Hepatic Chemistry Parameters: Change From Baseline/Screening Alkaline phosphatase	-1.00 U/L Interval -39.0 to 33.0	1.50 U/L Interval -68.0 to 35.0	-5.50 U/L Interval -70.0 to 21.0	0.00 U/L Interval -61.0 to 13.0	—
Hepatic Chemistry Parameters: Change From Baseline/Screening ALT	0.00 U/L Interval -21.0 to 27.0	2.50 U/L Interval -19.0 to 26.0	2.00 U/L Interval -21.0 to 35.0	2.00 U/L Interval -27.0 to 11.0	—
Hepatic Chemistry Parameters: Change From Baseline/Screening AST	1.00 U/L Interval -25.0 to 22.0	0.00 U/L Interval -23.0 to 9.0	1.00 U/L Interval -17.0 to 27.0	0.00 U/L Interval -10.0 to 7.0	—

SECONDARY outcome

Timeframe: Baseline to 24 months

Population: Subjects who participated in the blood draws with data available during each window period

Summary of changes in hepatic (total bilirubin) and renal (Blood urea nitrogen (BUN), creatinine) parameters from screening/baseline through each window period (Baseline to ≤6 Months, >6 Months to ≤12 Months, >12 Months to ≤18 Months, and >18 Months to ≤24 Months)

Outcome measures

Measure	Baseline n=52 Participants	Baseline to ≤6 Months n=34 Participants	>6 Months to ≤12 Months n=26 Participants	>12 Months to ≤18 Months n=15 Participants	>18 Months to ≤24 Months
Renal and Hepatic Chemistry Parameters: Change From Baseline/Screening BUN	0.65 mg/dL Interval -7.7 to 9.8	-0.15 mg/dL Interval -9.2 to 10.7	1.50 mg/dL Interval -8.4 to 7.7	-0.50 mg/dL Interval -4.3 to 9.0	—
Renal and Hepatic Chemistry Parameters: Change From Baseline/Screening Creatinine	0.02 mg/dL Interval -0.2 to 0.6	0.01 mg/dL Interval -0.1 to 0.3	0.04 mg/dL Interval -0.2 to 0.2	0.00 mg/dL Interval -0.2 to 0.2	—
Renal and Hepatic Chemistry Parameters: Change From Baseline/Screening Total bilirubin	0.00 mg/dL Interval -0.7 to 0.6	0.00 mg/dL Interval -0.5 to 0.5	0.00 mg/dL Interval -0.3 to 0.6	0.00 mg/dL Interval -0.5 to 0.5	—

SECONDARY outcome

Timeframe: Baseline to 24 Months

Population: Subjects who participated in the blood draws with data available during each window period

All IgG and IgM titers at screening were ≤ 4. A 2-dilution step increase was defined as follows: • The titer at each 6-month visit must be ≥ 4 when the screening titer = 0 • Each 6-month visit titer / screening titer should be ≥ 4. 6 month window periods are: baseline to ≤6 months, >6 months to ≤12 months, >12 months to ≤18 months, and >18 months to ≤24 months

Outcome measures

Measure	Baseline n=52 Participants	Baseline to ≤6 Months n=34 Participants	>6 Months to ≤12 Months n=26 Participants	>12 Months to ≤18 Months n=15 Participants	>18 Months to ≤24 Months
ARALAST Antibody Titers: Participants With at Least 2-Dilution Step Increases From Screening IgG	2 Participants	0 Participants	0 Participants	0 Participants	—
ARALAST Antibody Titers: Participants With at Least 2-Dilution Step Increases From Screening IgM	2 Participants	1 Participants	1 Participants	0 Participants	—

Adverse Events

Overall Study

Serious events: 39 serious events

Other events: 97 other events

Deaths: 0 deaths

Serious adverse events

Measure	Overall Study n=127 participants at risk
Cardiac disorders Bradycardia	0.79% 1/127 • Number of events 1 • Throughout the entire study period (2 years, 7 months)
Cardiac disorders Cardiac failure congestive	1.6% 2/127 • Number of events 2 • Throughout the entire study period (2 years, 7 months)
Cardiac disorders Cardio-respiratory arrest	0.79% 1/127 • Number of events 1 • Throughout the entire study period (2 years, 7 months)
Cardiac disorders Myocardial infarction	0.79% 1/127 • Number of events 1 • Throughout the entire study period (2 years, 7 months)
Gastrointestinal disorders Colon gangrene	0.79% 1/127 • Number of events 1 • Throughout the entire study period (2 years, 7 months)
Gastrointestinal disorders Gastric ulcer	0.79% 1/127 • Number of events 1 • Throughout the entire study period (2 years, 7 months)
Gastrointestinal disorders Vomiting	0.79% 1/127 • Number of events 1 • Throughout the entire study period (2 years, 7 months)
General disorders Chest pain	1.6% 2/127 • Number of events 2 • Throughout the entire study period (2 years, 7 months)
General disorders Fatigue	0.79% 1/127 • Number of events 1 • Throughout the entire study period (2 years, 7 months)
General disorders Hernia	1.6% 2/127 • Number of events 2 • Throughout the entire study period (2 years, 7 months)
Hepatobiliary disorders Hepatic failure	0.79% 1/127 • Number of events 1 • Throughout the entire study period (2 years, 7 months)
Infections and infestations Gastroenteritis	0.79% 1/127 • Number of events 1 • Throughout the entire study period (2 years, 7 months)
Infections and infestations Pneumonia	4.7% 6/127 • Number of events 7 • Throughout the entire study period (2 years, 7 months)
Infections and infestations Pseudomonas infection	0.79% 1/127 • Number of events 2 • Throughout the entire study period (2 years, 7 months)
Infections and infestations Respiratory tract infection	2.4% 3/127 • Number of events 3 • Throughout the entire study period (2 years, 7 months)
Injury, poisoning and procedural complications Road traffic accident	1.6% 2/127 • Number of events 2 • Throughout the entire study period (2 years, 7 months)
Musculoskeletal and connective tissue disorders Intervertebral disc protrusion	0.79% 1/127 • Number of events 1 • Throughout the entire study period (2 years, 7 months)
Musculoskeletal and connective tissue disorders Muscular weakness	0.79% 1/127 • Number of events 1 • Throughout the entire study period (2 years, 7 months)
Nervous system disorders Disorientation	1.6% 2/127 • Number of events 2 • Throughout the entire study period (2 years, 7 months)
Respiratory, thoracic and mediastinal disorders Chronic obstructive pulmonary disease	8.7% 11/127 • Number of events 13 • Throughout the entire study period (2 years, 7 months)
Respiratory, thoracic and mediastinal disorders Respiratory failure	3.9% 5/127 • Number of events 5 • Throughout the entire study period (2 years, 7 months)
Vascular disorders Peripheral artery aneurysm	0.79% 1/127 • Number of events 1 • Throughout the entire study period (2 years, 7 months)

Other adverse events

Measure	Overall Study n=127 participants at risk
Gastrointestinal disorders Diarrhea	5.5% 7/127 • Number of events 21 • Throughout the entire study period (2 years, 7 months)
General disorders Fatigue	11.8% 15/127 • Number of events 123 • Throughout the entire study period (2 years, 7 months)
General disorders Infusion related reaction	7.1% 9/127 • Number of events 13 • Throughout the entire study period (2 years, 7 months)
Immune system disorders Hypersensitivity	10.2% 13/127 • Number of events 28 • Throughout the entire study period (2 years, 7 months)
Infections and infestations Influenza	5.5% 7/127 • Number of events 27 • Throughout the entire study period (2 years, 7 months)
Infections and infestations Lower respiratory tract infection	18.9% 24/127 • Number of events 35 • Throughout the entire study period (2 years, 7 months)
Infections and infestations Oral fungal infection	7.9% 10/127 • Number of events 18 • Throughout the entire study period (2 years, 7 months)
Infections and infestations Respiratory tract infection	26.0% 33/127 • Number of events 66 • Throughout the entire study period (2 years, 7 months)
Infections and infestations Upper respiratory tract infection	22.0% 28/127 • Number of events 45 • Throughout the entire study period (2 years, 7 months)
Nervous system disorders Headache	18.1% 23/127 • Number of events 98 • Throughout the entire study period (2 years, 7 months)
Respiratory, thoracic and mediastinal disorders Chronic obstructive pulmonary disease	18.1% 23/127 • Number of events 33 • Throughout the entire study period (2 years, 7 months)
Surgical and medical procedures Elective procedure	6.3% 8/127 • Number of events 9 • Throughout the entire study period (2 years, 7 months)
Vascular disorders Poor venous access	6.3% 8/127 • Number of events 10 • Throughout the entire study period (2 years, 7 months)

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Email: ClinicalTransparency@shire.com

Results disclosure agreements

• Principal investigator is a sponsor employee Baxter's agreements with PIs vary per individual PI, but contain common elements. Baxter may require a review of results communications (e.g., for confidential information \& intellectual property) ≥45 days prior to submission or communication for review and comment. Baxter reserves the right to remove all confidential information from all publications.
• Publication restrictions are in place

Restriction type: OTHER