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Randomized controlled study of ECP with methoxsalen as first-line treatment of patients with moderate to severe cGVHD.

ABSTRACT: The investigation of extracorporeal photopheresis (ECP) plus standard of care (SoC) (SoC+ECP) in chronic graft-versus-host disease (cGVHD) within prospective, randomized clinical studies is limited, despite its frequent clinical use. This phase 1/pilot study was the first randomized, prospective study to investigate ECP use as first-line therapy in cGVHD, based on the 2015 National Institutes of Health (NIH) consensus criteria for diagnosis and response assessment. Adult patients with new-onset (?3 years of hematopoietic stem cell transplantation) moderate or severe cGVHD were randomized 1:1 to 26 weeks of SoC+ECP vs SoC (corticosteroids and cyclosporine A/tacrolimus) between 2011 and 2015. The primary endpoint was overall response rate (ORR), defined as complete or partial response, at week 28 in the intention-to-treat population (ITT). Other outcomes included quality of life (QoL) measures and safety. Sixty patients were randomized; ITT included 53 patients (SoC+ECP: 29; SoC: 24). Week 28 ORR was 74.1% (SoC+ECP) and 60.9% (SoC). Investigator-assessed ORR was 56.0% (SoC+ECP) and 66.7% (SoC). Patients treated with SoC experienced a decline in QoL over the 28-week study period; QoL remained unchanged in SoC+ECP patients. Most frequent treatment-emergent adverse events (TEAEs) in SoC+ECP patients were hypertension (31.0%), cough (20.7%), dyspnea (17.2%), and fatigue (17.2%). Seventeen patients (SoC+ECP: 8; SoC: 9) experienced 35 serious adverse events (SAEs). No TEAEs or SAEs were considered related to the ECP instrument or methoxsalen. The encouraging short-term results of this study could inform the design of subsequent studies. This trial was registered at www.clinicaltrials.gov as #NCT01380535.

SUBMITTER: Jagasia M

PROVIDER: S-EPMC6650730 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature

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Randomized controlled study of ECP with methoxsalen as first-line treatment of patients with moderate to severe cGVHD.

Jagasia Madan M Scheid Christof C Socié Gérard G Ayuk Francis Ayuketang FA Tischer Johanna J Donato Michele L ML Bátai Árpád Á Chen Heidi H Chen Sheau-Chiann SC Chin Thomas T Boodée Henri H Mitri Ghaith G Greinix Hildegard T HT

Blood advances 20190701 14

The investigation of extracorporeal photopheresis (ECP) plus standard of care (SoC) (SoC+ECP) in chronic graft-versus-host disease (cGVHD) within prospective, randomized clinical studies is limited, despite its frequent clinical use. This phase 1/pilot study was the first randomized, prospective study to investigate ECP use as first-line therapy in cGVHD, based on the 2015 National Institutes of Health (NIH) consensus criteria for diagnosis and response assessment. Adult patients with new-onset ...[more]

PMID: 31332045

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Randomized controlled study of ECP with methoxsalen as first-line treatment of patients with moderate to severe cGVHD.

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Randomized controlled study of ECP with methoxsalen as first-line treatment of patients with moderate to severe cGVHD.

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