Hematopoietic Stem Cell Transplant, Thrombotic Microangiopathy
Conditions
Keywords
Thrombotic Microangiopathy, TMA, Hematopoietic Stem Cell Transplant, HSCT, Observational Study, Real-world Data, Retrospective
Brief summary
The purpose of this study was to assess OS outcomes in pediatric (≥ 28 days of age at the time of HSCT-TMA diagnosis) and adult participants diagnosed with HSCT-TMA(referred to as TMA from hereon) within 52 weeks after HSCT and who were either complement inhibitor treatment naïve or had been treated with eculizumab.
Interventions
None listed
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
4 Weeks to No maximum
Healthy volunteers
No
Inclusion criteria
* Participant must be ≥ 28 days of age at the time of TMA diagnosis. * Body weight ≥ 5 kg at the time of TMA diagnosis. * Documented TMA on/after 01 Jan 2010 to 30 Jun 2024 with concurrent renal dysfunction with no documented alternative explanation ≤ 52 weeks from the HSCT. * Documentation of participant's vital status through 52 weeks after the date of TMA diagnosis. * Documentation of date and type of HSCT, date of TMA, absence of acute GVHD, and infection status at time of TMA diagnosis. * Documentation of at least 2 of the following: indication for most recent HSCT, serum creatinine at time of TMA diagnosis, presence of multiorgan dysfunction at time of TMA diagnosis. * Informed consent obtained if required by local regulations.
Exclusion criteria
* Medical Conditions (Ongoing at the Time of TMA Diagnosis): TTP, ST-HUS, Immune-mediated hemolysis not due to TMA, DIC, Bone marrow/graft failure of HSCT, VOD (regardless of severity), HIV infection, Sepsis that required vasopressor support. * Prior/Concomitant Therapy: Received a complement inhibitor other than eculizumab from time of suspicion of HSCT-TMA diagnosis through 52 weeks post-HSCT-TMA diagnosis.. * Other Exclusions: Participation in an investigational drug or device study for the treatment of TMA within 30 days prior to TMA diagnosis or during the 52 weeks following TMA diagnosis.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Survival (OS) | 52 weeks after TMA diagnosis | To assess OS through 52 weeks after TMA diagnosis. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Overall Survival (OS) | 100 days and 26 weeks after TMA diagnosis | OS through 100 days and 26 weeks after TMA diagnosis. |
| Non-relapse mortality | Through 100 days, 26 weeks, and 52 weeks after TMA diagnosis | Non-relapse mortality, defined as death due to any cause other than primary disease progression (i.e. indication fror transplant) or relapse. |
Countries
Belgium, Brazil, France, Greece, Italy, Japan, South Korea, Spain, United Kingdom, United States
Outcome results
None listed