Stem Cell Transplant Complications, Thrombotic Microangiopathies
Conditions
Keywords
Hematopoietic Stem Cell Transplant, Transplant-associated TMA, Thrombotic Microangiopathy (TMA)
Brief summary
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.
Interventions
OTHERNo intervention
No study intervention will be administered as part of this study.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Observational model
CASE_ONLY
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
1 Months to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of patients achieving TMA Response | During the 26-week period after HSCT-TMA diagnosis | 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) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of patients achieving TMA response | During the 52-week period after HSCT-TMA diagnosis | — |
| Changes in individual components of TMA response | From baseline to 26 weeks and to 52 weeks after HSCT-TMA diagnosis | Platelets, lactate dehydrogenase, eGFR, chronic kidney disease stage, dialysis status |
| Overall survival | At 26 weeks and 52 weeks after HSCT-TMA diagnosis | — |
| Nonrelapse mortality | At 26 weeks and 52 weeks after HSCT-TMA diagnosis | Death due to any cause during the study, with the exception of death due to underlying disease progression or relapse |
Countries
United States
Outcome results
None listed