Study in Adult and Pediatric Patients With HSCT-TMA

Alexion Pharmaceuticals, Inc.

Overview

This is an observational, retrospective study designed to assess outcomes in patients diagnosed with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) who were not treated with complement component (C5) inhibitor therapy. Data required to evaluate study outcomes will be abstracted from the medical records of all patients who meet study eligibility criteria.

Study Type

OBSERVATIONAL

Conditions

Stem Cell Transplant Complications Thrombotic Microangiopathies

Interventions

No interventionOTHER

No study intervention will be administered as part of this study.

Eligibility

Sex: ALLMin age: 1 Month

Medical Language ↔ Plain English

Inclusion Criteria: * Body weight ≥ 5 kg at the time of HSCT-TMA diagnosis * Documented TMA diagnosis within 6 months from the HSCT * Evidence of renal dysfunction * Presence of hypertension Exclusion Criteria: * History or presence of familial or acquired 'a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13' (ADAMTS13) deficiency (activity \< 5%) * Shiga toxin-related hemolytic uremic syndrome (ST-HUS) * Positive direct Coombs test * Diagnosis of disseminated intravascular coagulation * History or presence of bone marrow/graft failure * Diagnosis of veno-occlusive disease * Received a complement inhibitor (eg, eculizumab) post-HSCT through 12 months post TMA diagnosis

Locations (1)

Clinical Trial Site

Salt Lake City, Utah, United States

Outcomes

Primary Outcomes

Proportion of patients achieving TMA Response

TMA Response is defined as platelet count ≥ 50,000/mm\^3, lactate dehydrogenase \< 1.5 upper limit of normal, absence of schistocytes (if present at baseline), and increase in eGFR ≥ 30% from baseline or discontinuation of dialysis (for patients on dialysis at baseline)

Time frame: During the 26-week period after HSCT-TMA diagnosis

Secondary Outcomes

Proportion of patients achieving TMA response

Time frame: During the 52-week period after HSCT-TMA diagnosis

Changes in individual components of TMA response

Platelets, lactate dehydrogenase, eGFR, chronic kidney disease stage, dialysis status

Time frame: From baseline to 26 weeks and to 52 weeks after HSCT-TMA diagnosis

Overall survival

Time frame: At 26 weeks and 52 weeks after HSCT-TMA diagnosis

Nonrelapse mortality

Death due to any cause during the study, with the exception of death due to underlying disease progression or relapse

Time frame: At 26 weeks and 52 weeks after HSCT-TMA diagnosis

Data from ClinicalTrials.gov

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