Zastosowanie CXCL9 jako biomarkera skuteczności Acthar (Acthar)

Eksperymentalny: Żel Acthar

Po 4-tygodniowym okresie monitorowania stanu wyjściowego żel Acthar zostanie podany we wstrzyknięciu domięśniowym lub podskórnym. Początkowe dawkowanie będzie wynosić 40 U co 72 godziny (lub dwa razy w tygodniu) przez 4 tygodnie. Dawka zostanie następnie zwiększona do 80 U z podobną częstotliwością przez 8 tygodni i do 16 tygodni.

Lek: Żel Acthar

Żel Acthar podany IM lub SQ zgodnie z ulotką dołączoną do opakowania

Inne nazwy:

• Akthar

Number of Participants That Show a Decrease in Blood CXCL9 Levels by 50%

Quantitative polymerase chain reaction assay was used to measure the gene expression for the gene CXCL9 using whole blood samples. The change in cycle threshold from before starting medication to the end of treatment was calculated. From this calculation, the number of participants that met the criteria of a 50% or greater drop in CXCL9 levels is reported.

Participants With Improvement in Forced Vital Capacity (FVC) Liters by 5 Percent or More

Participants With Improvement in Dyspnea Score

The University of California San Diego Shortness of Breath Questionnaire (UCSD SOBQ) is a 24-item patient-reported outcome measure that assesses the severity of dyspnea during activities of daily living. Participants rate shortness of breath for each activity on a 6-point scale from 0 ("not at all") to 5 ("maximally or unable to do because of breathlessness"). Item scores are summed to generate a total score ranging from 0 to 120. Higher total scores represent worse dyspnea severity, while lower scores indicate less dyspnea. The SOBQ does not contain separate subscales; all 24 items are equally weighted and combined by simple summation to calculate the total score. If a participant does not routinely perform an activity, they are instructed to estimate anticipated dyspnea, minimizing missing data.