A prospective, single-blind, pilot study examining the use of Acthar in patients with advanced pulmonary sarcoidosis1
Significant reductions in steroid dosages and improvements in pulmonary function were associated with Acthar treatment
A MULTICENTER, PROSPECTIVE, SINGLE-BLIND STUDY IN ADVANCED PULMONARY SARCOIDOSIS PATIENTS RECEIVING PREDNISONE (n=16)1
|PRIMARY||Evaluated prednisone-sparing effect of Acthar|
|OTHER||Evaluated the effect of Acthar on pulmonary function, chest imaging, and health-related quality-of-life|
SELECTED INCLUSION CRITERIA
- All patients met American Thoracic Society criteria for diagnosis
- On a stable dose of ≥5 mg prednisone for ≥3 months
- Deterioration of pulmonary disease as defined by a decrease of 5% forced vital capacity (FVC) in the previous year
SELECTED EXCLUSION CRITERIA
- Treatment with anti-TNF antibody (eg, infliximab, adalimumab) in prior 6 months
- Receiving treatment for sarcoidosis-associated pulmonary hypertension
Significantly reduced the prednisone dose
When the effect of Acthar at any dose was analyzed, there was a significant reduction in the prednisone dosage at 7 weeks (P=.0156) and 24 weeks (P=.0078) compared to the initial dosage of prednisone.1
9 OUT OF 16 PATIENTS reduced their prednisone dose by at least 50%1
3 OF THOSE 9 PATIENTS stopped taking prednisone by week 241,2
INITIAL AND FINAL PREDNISONE DAILY DOSAGES PER PATIENT (n=16) AT WEEK 241,2
*Per protocol, Acthar dose was halved due to toxicity.
Of patients enrolled, 16 of 18 completed the 24-week study. One patient withdrew prior to starting therapy and 1 withdrew due to toxicity.
At week 7, there was no significant difference in reduction of prednisone dosage for those receiving 80 units of Acthar (median 0 mg, range 0 to -20 mg) vs those receiving 40 units of Acthar (median -2.75 mg, range 0 to -12.5 mg, P >.05)1
Additional endpoints: Patient-reported outcomes1
SUMMARY OF CHANGES IN QUALITY-OF-LIFE MEASURES DURING ACTHAR THERAPY1
‡Compared to week 0, P =.0043.
§Compared to week 0, P =.0084.
∥Compared to week 0, P =.0034.
¶Compared to week 0, P =.0067.
#Compared to week 0, P =.0107.
**Compared to week 0, P =.0067.
- There were significant differences in the KSQ on several domains, including general health status (GHS), general health status lung (GHS lung), and lung
- For all patients, there was a significant improvement (rise) in GHS at weeks 7 and 24 vs week 0. There was no difference in GHS change between the 40- and 80-unit treatment groups
- Neither the SGRQ total nor any of its 3 components changed significantly during the study
- There was a significant fall (less fatigue) in the FAS score at week 24 (P =.0067)
- At week 7, 10 patients had a 4-point or greater drop in their FAS score
- By week 24, 8 patients still had a 4-point or greater drop in their FAS score
94% of patients (16 of 17) were able to remain on Acthar for the full 24 weeks of the study period.1
Additional endpoints: DLCO and SUV
Significant changes in DLCO1
- In the 14 patients who had their DLCO measured prior to and after 24 weeks of therapy, there was a significant rise in the DLCO percent predicted after 24 weeks
No change in FVC percent predicted1
- No significant reduction in FVC percent predicted between the 2 dosage groups after 7 weeks of therapy
SUV of highest lung lesion, as measured by FDG-PET scans at week 24, fell from a median 4.0 to 2.9 (P =.0085)1
- There was no correlation between PET scan results and the change in DLCO, FVC percent predicted, or dose of prednisone during the 24 weeks of the study
- There was no significant difference in reduction of prednisone dosage, changes in FVC percent predicted, or the quality-of-life measures of the patients between the 2 dosage groups at week 7
DLCO=diffusing capacity of the lungs for carbon monoxide. FDG-PET=F-fluorodeoxyglucose positron emission tomography. SUV=standardized update value.
- 1 withdrew during the initial loading period
- 8 patients complained of one or more of the following: jitteriness (n=6), headache (n=3), edema (n=2), and nausea (n=1)
- 5 stopped loading dose between days 7 and 9 due to toxicity but began the randomized phase
- 1 stopped treatment within 2 weeks because of toxicity in the randomized phase in the 40-U arm
- 7 reduced their dose by half (2 in the 40-U arm and 5 in the 80-U arm, P >.05)
- 8 complained of anxiety and fluid retention on the day of drug administration; many of these occurred during the daily loading doses
- Adverse events associated with Acthar use were collected prospectively for all patients
- No significant differences in reported toxicity, including changes in moodiness, appetite, or bruising
- No significant change in weight during the 24-week study period
- Of the 6 patients who had elevated hemoglobin A1c levels at baseline, none of the values fell into the normal range by the end of 24 weeks
- There were no changes in the patients’ diabetic or hypertensive medications during the course of the study