Prospective Outcomes of Second-Line Therapy in Acute Graft-Versus-Host Study Including ECP (POSTAGE) (POSTAGE)

This research trial studies medical chart review in determining outcomes of second-line therapy in patients with acute graft-versus-host disease previously treated with extracorporeal photopheresis or other systemic therapies. Gathering information about second-line therapy in patients with acute graft-versus-host disease may help doctors learn more about the disease and find better treatment.

Study Overview

• Status: Terminated
• Conditions: Graft Versus Host Disease
• Intervention / Treatment: Other: medical chart review; Other: quality-of-life assessment

Detailed Description

PRIMARY OBJECTIVES:

I. To determine 6-month freedom from treatment failure for second-line therapy for acute graft versus host disease (aGVHD).

II. To show that extracorporeal photopheresis (ECP) is associated with a superior 6 month (m) freedom from treatment failure (FFTF) as compared to other treatment modalities for second line therapy for aGVHD.

III. To describe health care burden in patients receiving second line therapy for acute GVHD.

IV. Quality of life measurement using Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) in patients receiving second line therapy for aGVHD.

OUTLINE:

Study data are collected and managed using Research Electronic Data Capture (REDCap) tools at baseline and on days 5, 28, and 56.

After completion of study, patients are followed up at 6 months, and 1 and 2 years.

• Study Type: Observational
• Enrollment (Actual): 19

Contacts and Locations

Study Locations

• Austria
  • Vienna, Austria, A-1090
    • Medical University Vienna
• Germany
  • Regensburg, Germany, 93053
    • Klinikum der Universität Regensburg
• United Kingdom
  • Nottingham, United Kingdom, NG5 1PB
    • Nottingham City Hospital
• United States
  • Arizona
    • Scottsdale, Arizona, United States, 85259
      • Mayo Clinic in Arizona
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • The Cleveland Clinic
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt-Ingram Cancer Center (VICC)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

This study targets patients with acute graft-versus-host disease previously treated with extracorporeal photopheresis or other systemic therapies. Patients can be enrolled by all participating locations.

Description

Inclusion Criteria:

• Enrollment on study is within 5 days (including weekends) of starting second-line therapy
• aGVHD grade 2 or higher at time of enrollment; patients with late acute and recurrent aGVHD are permitted
• Donor lymphocyte induced aGVHD is permitted ONLY IF donor lymphocyte infusion given for mixed chimerism and not for progression of disease and meeting all other inclusion and exclusion criteria will be eligible

• Corticosteroid refractory or corticosteroid dependent aGVHD

  • Corticosteroid refractory aGVHD is defined as worsening of aGVHD after 3 days of systemic corticosteroids (minimum dose of 1 mg/kg), or no improvement after 7 days of systemic corticosteroids (minimum dose of 1 mg/kg)
  • Corticosteroid dependent aGVHD is defined as recurrence of aGVHD (grade 2 or higher) during corticosteroid taper and prior to reaching 50% of initial starting dose of corticosteroids
• Informed consent form

Exclusion Criteria:

• Has received corticosteroids at 2 mg/kg or higher for 3 weeks or longer as part of first-line therapy for aGVHD
• Has received systemic therapy other than corticosteroids for treatment of aGVHD as part of first-line therapy for acute GVHD; simultaneous uses of topical or enteric corticosteroids or psoralen plus ultraviolet A (PUVA) for first-line are permitted
• aGVHD after second hematopoietic cell transplantation (HCT) is excluded
• Karnofsky performance status =< 50%
• Requiring mechanical ventilation or renal replacement therapy at the time of enrollment
• Histologic or flow-cytometric evidence of relapse or progression of underlying disease; molecular or cytogenetic presence of disease is permitted; mixed chimerism is permitted
• Current or prior diagnosis of chronic GVHD (classic or overlap) as defined by National Institutes of Health (NIH) consensus criteria
• Donor lymphocyte infusion (DLI)-induced aGVHD when DLI was given for progression of the underlying disease

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Observational (medical chart review); Study data are collected and managed using REDCap tools at baseline and on days 5, 28, and 56.	Other: medical chart review; Ancillary studies; Other Names: chart review; Other: quality-of-life assessment; Ancillary studies; Other Names: quality of life assessment

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
6-month freedom from treatment failure, defined as a patient being alive, without relapse of underlying disease, and without the addition of new systemic therapy for the treatment of aGVHD, within 6 months of starting second line therapy	Rates of 6 m FFTF will be estimated by subtracting rates of total failures from 100%.	6 months
Cumulative incidence estimates of relapse	Cox regression models will be used to identify risk factors for failure.	Up to 2 years
Cumulative incidence estimates of non-relapse mortality	Cox regression models will be used to identify risk factors for failure.	Up to 2 years
Treatment change as causes of failure during second line treatment	Cox regression models will be used to identify risk factors for failure.	Up to 2 years
Health care burden in patients receiving second line therapy for acute GVHD	The incremental budget spend between the patients receiving treatment with ECP and patients receiving other modalities will be measured and economic methods will be used to calculate an incremental cost effectiveness ratio for relevant clinical end points. Health economic data relating to hospitalizations (length of stay in myelosuppressive unit, high-dependency unit [step-down unit], intensive care unit), high cost drugs, total parenteral nutrition and surgical procedures will also be collected allowing comparisons to be made between patients treated with ECP and other treatment options.	Up to 6 months
Quality of life measured using FACT-BMT		Up to 6 months

Collaborators and Investigators

• Sponsor: Vanderbilt-Ingram Cancer Center
• Collaborators: National Cancer Institute (NCI)
• Investigators: Principal Investigator: Madan Jagasia, Vanderbilt-Ingram Cancer Center

Publications and helpful links

Helpful Links

• Vanderbilt-Ingram Cancer Center, Find a Clinical Trial

Study record dates

Study Major Dates

• Study Start (Actual): August 29, 2014
• Primary Completion (Actual): October 11, 2018
• Study Completion (Actual): October 11, 2018

Study Registration Dates

• First Submitted: May 28, 2014
• First Submitted That Met QC Criteria: May 28, 2014
• First Posted (Estimate): May 30, 2014

Study Record Updates

• Last Update Posted (Actual): October 17, 2019
• Last Update Submitted That Met QC Criteria: October 14, 2019
• Last Verified: October 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: VICC BMT 1401 (Other Identifier: Vanderbilt-Ingram Cancer Center); P30CA068485 (U.S. NIH Grant/Contract); NCI-2014-01026 (Registry Identifier: CTRP (Clinical Trial Reporting Program))

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No