A Study of Patients With Pure Red Cell Aplasia Associated With Recombinant Human Erythropoietin Treatment

Study Overview
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Brief Summary
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The purpose of this study is to investigate the relationship of anti-erythropoietin antibodies to the clinical course and outcome of pure red cell aplasia (PRCA) in participants currently or previously treated with recombinant human erythropoietin.

Detailed Description
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This is a multicenter (study conducted at multiple sites), observational (study in which the investigators/physicians observe the participant's data and measure their outcomes) study. Approximately 150 participants will be enrolled in this study. The study consists of an initial observation phase and extended observation period. An initial observation phase starting at enrollment and ending when 24 months have elapsed since the date of loss of efficacy (LOE), supplemented with retrospective data collection for the period between LOE date and date of enrollment in the study. Participants remaining epoetin alfa (EPO-Ab) positive 24 months after LOE will enter a 2-year extended observation period. Study visits will take place every month during the initial observation phase and data will be collected every 6 months during the extended observation phase. Safety evaluations will include assessment of adverse events, clinical laboratory tests, vital signs, and physical examination which will be monitored throughout the study. The total study duration for each participant will be approximately for 4 years.

Official Title
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Observational, Multicenter Study of Subjects With Pure Red Cell Aplasia Associated With r-HuEPO Treatment

Conditions
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Pure Red-cell Aplasia

Intervention / Treatment
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* Other: No intervention


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Pure red cell aplasia (PRCA) associated with recombinant human erythropoietin (r-HuEPO) treatment Anemia unresponsive to r-HuEPO treatment PRCA associated with erythropoietin treatment followed by a sudden decrease (more than or equal to 2 gram per deciliter within 30 days) in a previously stable hemoglobin level Exclusion criteria: - Participants who are not fulfilling the inclusion criteria
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Pure Red Cell Aplasia (PRCA)<br>This study will examine the relationship of the presence of anti-erythropoietin antibodies to the clinical course and outcome of participants currently or previously treated with recombinant human erythropoietin and who have PRCA identified from all notified reports (spontaneous postmarketing reports or from clinical trials reports). | Other: No intervention<br>* This is an observational study. No medication will be given to the participants. Participants will receive standard-of-care treatment from their individual physicians.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with Pure Red Cell Aplasia (PRCA) outcome (Initial observation phase) | The PRCA outcome is measured by anti-epoetin alfa qualitative test. Persistence of PRCA is defined as: 1) absolute reticulocyte count less than 30,000 per cubic millimeter; and/or 2) no reversal of erythroblastopenia on repeated bone marrow testing. Resolution of PRCA is defined as: 1) absolute reticulocyte count greater than or equal to 30,000 per cubic millimeter; and/or 2) reversal of erythroblastopenia on repeated bone marrow testing. | Up to 24 months after the date of loss of efficacy | 
| Number of participants with pure red cell aplasia outcome (Extended observation phase) | Participants remaining anti-epoetin alfa positive 24 months after loss of efficacy will enter in the extended observation period. | Up to 2 years after the enrollment in the extended observation phase | 
| Overall clinical outcome of pure red cell aplasia (Initial observation phase) | The overall clinical outcome is evaluated by anti-epoetin alfa qualitative test. Overall clinical status will be recorded at each visit in the initial and extended observation phases using a categorical scale (improved, same, worsened, death). In case of death, the date and cause of death along with the the date and cause of death will be recorded. | Up to 24 months after the date of loss of efficacy | 
| Overall clinical outcome of pure red cell aplasia (Extended observation phase) |  | Up to 2 years after the enrollment in the extended observation phase | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Different treatment modalities with pure red cell aplasia outcome (Initial observation phase) |  | Up to 24 months after the date of loss of efficacy | 
| Different treatment modalities with pure red cell aplasia outcome (Extended observation phase) |  | Up to 2 years after the enrollment in the extended observation phase | 
| Risk factors for Loss of Efficacy (LOE) and pure red cell aplasia (PRCA) outcome | This data will be collected retrospectively and the date of LOE will be determined by the sponsor based upon reported data. PRCA duration groups will be summarized by potential risk factors to evaluate the relationship of risk factors to the duration of PRCA. | Period between LOE date and date of enrollment in the study | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pure red-cell aplasia, Anemia, Red blood cells, Epoetin alfa (Eprex), Anti-erythropoietin antibodies, Recombinant human erythropoietin