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

Terminated

• Conditions: Graft Versus Host Disease
• Interventions: Other: medical chart review; Other: quality-of-life assessment

• Registration Number: NCT02151539
• Lead Sponsor: Vanderbilt-Ingram Cancer Center

Brief Summary

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.

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 Timeline

• Study First Submitted: 2014-05-28
• Study First Submitted QC: 2014-05-28
• Study First Posted: 2014-05-30
• Study Start: 2014-08-29
• Primary Completion: 2018-10-11
• Study Completion: 2018-10-11
• Status Verified: 2019-10
• Last Update Submitted: 2019-10-14
• Last Update Posted: 2019-10-17

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 19

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Observational (medical chart review)	quality-of-life assessment	Study data are collected and managed using REDCap tools at baseline and on days 5, 28, and 56.
Observational (medical chart review)	medical chart review	Study data are collected and managed using REDCap tools at baseline and on days 5, 28, and 56.

Primary Outcome Measures

Name	Time	Method
Quality of life measured using FACT-BMT	Up to 6 months
Cumulative incidence estimates of relapse	Up to 2 years	Cox regression models will be used to identify risk factors for failure.
Cumulative incidence estimates of non-relapse mortality	Up to 2 years	Cox regression models will be used to identify risk factors for failure.
Treatment change as causes of failure during second line treatment	Up to 2 years	Cox regression models will be used to identify risk factors for failure.
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	6 months	Rates of 6 m FFTF will be estimated by subtracting rates of total failures from 100%.
Health care burden in patients receiving second line therapy for acute GVHD	Up to 6 months	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.

Trial Locations

Locations (7)

Mayo Clinic in Arizona; 🇺🇸 Scottsdale, Arizona, United States

Klinikum der Universität Regensburg; 🇩🇪 Regensburg, Germany

Nottingham City Hospital; 🇬🇧 Nottingham, United Kingdom

The Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States

Vanderbilt-Ingram Cancer Center (VICC); 🇺🇸 Nashville, Tennessee, United States

Medical University Vienna; 🇦🇹 Vienna, Austria

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States