Study in Adult and Pediatric Patients With HSCT-TMA

Study Overview
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Brief Summary
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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.

Official Title
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A Retrospective Observational Study of Adult and Pediatric Patients With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplant (HSCT)

Conditions
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Stem Cell Transplant Complications, Thrombotic Microangiopathies

Intervention / Treatment
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* Other: No intervention


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 1 Month

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 |
| --- | --- |
| Patients Diagnosed with HSCT-TMA<br> | Other: No intervention<br>* No study intervention will be administered as part of this study.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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) | During the 26-week period after HSCT-TMA diagnosis | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of patients achieving TMA response |  | 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 | From baseline to 26 weeks and to 52 weeks after HSCT-TMA diagnosis | 
| Overall survival |  | 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 | At 26 weeks and 52 weeks after HSCT-TMA diagnosis | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hematopoietic Stem Cell Transplant, Transplant-associated TMA, Thrombotic Microangiopathy (TMA)