Premium
Patient‐reported outcomes in relapsed/refractory multiple myeloma treated with melflufen plus dexamethasone: analyses from the Phase II HORIZON study
Author(s) -
Larocca Alessandra,
Leleu Xavier,
Touzeau Cyrille,
Bladé Joan,
Paner Agne,
Mateos MaríaVictoria,
Cavo Michele,
Maisel Christopher,
Alegre Adrían,
Oriol Albert,
Raptis Anastasios,
RodriguezOtero Paula,
Mazumder Amitabha,
Laubach Jacob,
Nadeem Omar,
Sandberg Anna,
Orre Marie,
Torrång Anna,
Bakker Nicolaas A.,
Richardson Paul G.
Publication year - 2022
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.17887
Subject(s) - medicine , refractory (planetary science) , quality of life (healthcare) , dexamethasone , disease , multiple myeloma , clinical trial , physical therapy , intensive care medicine , nursing , physics , astrobiology
Summary Relapsed/refractory multiple myeloma (RRMM) is known to have a high burden of disease and complications associated with refractoriness to prior lines of therapy. Severe pain and fatigue symptoms and impairments in physical and emotional functioning have been strongly linked to reduced health‐related quality of life (HRQoL) in patients with RRMM. Assessment of patient reported‐outcome measures from the pivotal, Phase II HORIZON study (OP‐106; NCT02963493) in patients with RRMM ( n = 64) demonstrated that melphalan flufenamide (melflufen) plus dexamethasone treatment preserved HRQoL. Patients had clinically meaningful improvements, even after eight treatment cycles, in relevant scales such as global health status/QoL, physical functioning, emotional functioning, pain, and fatigue. Patients with triple‐class–refractory disease ( n = 50) displayed similar improvements. Patient‐reported outcome deterioration was delayed for a substantial amount of time in patients who experienced a response to melflufen plus dexamethasone treatment relative to patients who did not experience a response. These findings support the notion that treatment with melflufen plus dexamethasone may sustain or improve HRQoL over time in patients with RRMM, including in patients with triple‐class–refractory disease for whom outcomes are generally worse. The clinical benefits observed in patients from the HORIZON trial are encouraging and supportive of translation into real‐world practice.