PMID- 36965105
OWN - NLM
STAT- MEDLINE
DCOM- 20230620
LR  - 20230620
IS  - 1600-0609 (Electronic)
IS  - 0902-4441 (Linking)
VI  - 111
IP  - 1
DP  - 2023 Jul
TI  - Fatigue and health-related quality of life in paroxysmal nocturnal 
      haemoglobinuria: A post hoc analysis of the pegcetacoplan PEGASUS trial data.
PG  - 72-83
LID - 10.1111/ejh.13969 [doi]
AB  - OBJECTIVES: Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, non-malignant 
      haematological disorder associated with disabling fatigue and reduced 
      health-related quality of life. Post hoc analysis of PEGASUS phase 3 trial 
      (NCT03500549) characterised improvements in patient-reported fatigue measured by 
      functional assessment of chronic illness therapy-fatigue (FACIT-fatigue) 
      instrument item-level ratings for pegcetacoplan and eculizumab for the treatment 
      of PNH. METHODS: Item-level responder analysis was conducted on a >/=2-level change 
      from baseline (CFB) clinically important response (CIR) for the FACIT-fatigue 13 
      individual items rated on a 5-level Likert scale. We evaluated >/=2-level change 
      against the minimal clinically important difference (MCID) of the FACIT-fatigue 
      total score (>/=5 points) and clinical parameters, haemoglobin (Hb; >/=1 g/dL) and 
      normalised absolute reticulocyte count (ARC; 30-100 pg/cells). Logistic 
      regressions estimated baseline-to-Week-16 FACIT-fatigue item-level transitional 
      probabilities; Kaplan-Meier analysis estimated time to FACIT-fatigue item CIR. 
      RESULTS: Pegcetacoplan versus eculizumab was associated with significantly 
      greater odds of Week 16 CIR across 8/13 items and on total score MCID (OR [CI] 
      = 11.19 [3.73, 33.57]) and faster times to responses. The item-level CIR 
      threshold also showed clinical relevance on Hb level and ARC normalization. 
      CONCLUSIONS: Compared with eculizumab, pegcetacoplan was associated with 
      clinically meaningful greater improvements on a majority of FACIT-fatigue items.
CI  - (c) 2023 The Authors. European Journal of Haematology published by John Wiley & 
      Sons Ltd.
FAU - Panse, Jens
AU  - Panse J
AUID- ORCID: 0000-0001-6316-3112
AD  - Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, 
      University Hospital RWTH Aachen, Aachen, Germany.
AD  - Center for Integrated Oncology Aachen, Bonn, Cologne, Duesseldorf (CIO ABCD), 
      Aachen, Germany.
FAU - Wilson, Koo
AU  - Wilson K
AD  - Swedish Orphan Biovitrum AB, Stockholm, Sweden.
FAU - Fishman, Jesse
AU  - Fishman J
AD  - Apellis Pharmaceuticals, Inc., Waltham, Massachusetts, USA.
FAU - Wojciechowski, Piotr
AU  - Wojciechowski P
AD  - Assignity Sp. z O.O., Krakow, Poland.
FAU - Wdowiak, Marlena
AU  - Wdowiak M
AD  - Putnam Associates, Krakow, Poland.
FAU - Horneff, Regina
AU  - Horneff R
AD  - Swedish Orphan Biovitrum AB, Stockholm, Sweden.
FAU - Patriquin, Christopher J
AU  - Patriquin CJ
AD  - Division of Hematology, University Health Network, Toronto, Canada.
FAU - Oliver, Monika
AU  - Oliver M
AD  - Division of Hematology, University of Alberta, Alberta, Canada.
FAU - Hakimi, Zalmai
AU  - Hakimi Z
AD  - Swedish Orphan Biovitrum AB, Stockholm, Sweden.
LA  - eng
GR  - Swedish Orphan Biovitrum/
PT  - Clinical Trial, Phase III
PT  - Journal Article
DEP - 20230419
PL  - England
TA  - Eur J Haematol
JT  - European journal of haematology
JID - 8703985
RN  - 0 (Hemoglobins)
RN  - TO3JYR3BOU (pegcetacoplan)
SB  - IM
MH  - Humans
MH  - Fatigue/diagnosis/drug therapy/etiology
MH  - Hemoglobins
MH  - *Hemoglobinuria, Paroxysmal/diagnosis/drug therapy/pathology
MH  - Quality of Life
OTO - NOTNLM
OT  - FACIT-fatigue
OT  - PEGASUS
OT  - functional assessment of chronic illness therapy-fatigue
OT  - health related quality of life
OT  - paroxysmal nocturnal haemoglobinuria
EDAT- 2023/03/26 06:00
MHDA- 2023/06/09 06:42
CRDT- 2023/03/25 16:12
PHST- 2023/03/20 00:00 [revised]
PHST- 2023/01/04 00:00 [received]
PHST- 2023/03/22 00:00 [accepted]
PHST- 2023/06/09 06:42 [medline]
PHST- 2023/03/26 06:00 [pubmed]
PHST- 2023/03/25 16:12 [entrez]
AID - 10.1111/ejh.13969 [doi]
PST - ppublish
SO  - Eur J Haematol. 2023 Jul;111(1):72-83. doi: 10.1111/ejh.13969. Epub 2023 Apr 19.