Clinically important change for the FACIT-Fatigue scale in paroxysmal nocturnal hemoglobinuria: a derivation from international PNH registry patient data

Journal of Patient-Reported Outcomes

Table 1 Patient demographics and baseline disease characteristics

Characteristic	Patients (N = 423)
Sex, n (%)
Female	226 (53)
Male	197 (47)
Race, n (%)
White or Caucasian descent	355 (84)
Asian	51 (12)
Black or African descent	9 (2)
Other (unlisted, multiple races, Aboriginal)	8 (2)
Age at PNH start,^a year
Mean ± SD	39.0 ± 17.5
Median (Q1, Q3)	35.0 (23.2, 51.6)
Age at baseline,^b year
Mean ± SD	45.9 ± 17.2
Median (Q1, Q3)	44.4 (31.7, 60.2)
Mean ± SD baseline hemoglobin,^c g/dL	9.7 ± 2.1
Mean baseline LDH ratio × ULN,^d n (%)
< 1.5	40 (11)
≥ 1.5	324 (89)
Percent GPI-deficient granulocytes,^e n (%)
< 10%	5 (2)
≥ 10% to < 50%	35 (11)
≥ 50%	282 (88)
Physician-documented fatigue,^f n (%)
Yes	386 (93)
No	31 (7)
Mean ± SD baseline FACIT-Fatigue score	29.4 ± 12.9

FACIT functional assessment of chronic illness therapy, GPI glycosylphosphatidylinositol, LDH lactate dehydrogenase, PNH paroxysmal nocturnal hemoglobinuria, ULN upper limit of normal
^aPNH start date is defined as the earliest date among the following: PNH diagnosis, date of first PNH symptoms, and/or date of reported granulocytes clone lab text
^bBaseline was defined as the date of eculizumab initiation
^cBaseline hemoglobin n = 370
^dBaseline LDH n = 364
^eGPI = deficient granulocytes, n = 322
^fPhysician-documented fatigue, n = 417. Values are mean (%) unless otherwise noted