Hematopoietic Stem Cell Transplantation (HSCT), Hemosiderosis, Iron Overload, Liver Diseases
Conditions
Keywords
Iron overload, Liver fibrosis, Elastography, Long-term complications, Allogeneic hematopoietic stem-cell transplantation, graft-versus-host disease
Brief summary
This single-center, non interventional cohort study investigates whether chronic iron overload influences the incidence of liver complications in adults who are at least 10 years beyond allogeneic hematopoietic stem cell transplantation (allo HSCT). Approximately 400-500 survivors transplanted at Hôpital Saint Louis between January 2004 and December 2014 will be evaluated. Transplant characteristics, prior iron overload therapy, and historical hepatic events will be collected through the Promise database. At the same time, the prospective visit will include laboratory panels and non invasive liver stiffness measurement by FibroScan or shear wave elastography. The study's primary objective is to assess the impact of iron overload on the incidence of hepatic complications in patients more than 10 years after an allogeneic hematopoietic stem cell transplantation. Secondary aims include describing the spectrum and frequency of hepatic complications, determining risk factors (including graft versus host disease, conditioning regimen, and comorbidities), and evaluating the long term effectiveness of previous iron reduction treatments (phlebotomy or chelation). Results will clarify whether monitoring and treating iron overload in long term allo HSCT survivors can prevent late hepatic morbidity.
Interventions
OTHERCohort
Participants undergo one routine visit blood draw and non invasive liver stiffness measurement. Retrospective data are abstracted from medical records.
Sponsors
Assistance Publique - Hôpitaux de Paris
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Age ≥ 18 years at enrollment * Allogeneic HSCT performed at Hôpital Saint Louis between 2004/01/01 and 2014/12/31 * Alive and attending routine annual follow up within the two years of the study * Having given his non-opposition to study after understand overall aims * With health insurance coverage * Follow up consultation at Saint-Louis Hospital
Exclusion criteria
* Patient under legal protection (protection of the court, or in curatorship or guardianship). not in relapse of the hematological disease at the time of inclusion. • Patients under 45 Kg
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Event free survival | Up to 24 months | From any hepatic complication ≥ 10 years after allogeneic HSCT |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Overall free survival of any hepatic complication ≥ 10 years after HSCT | Up to 24 months | Survival without hepatic complication |
| Event free survival from liver fibrosis ≥ 10 years after HSCT | Up to 24 months | Time from HSCT to first diagnosis of liver fibrosis by elastography (FibroScan® ≥ 8 kPa or shear wave ≥ 7 kPa). baseline to fibrosis diagnosis |
| Event free survival for each specific hepatic complication ≥ 10 years after HSCT | Up to 24 months | Separate analyses for cirrhosis, portal hypertension, hepatic GVHD, viral hepatitis, steatosis, etc |
| Event free survival from iron overload ≥ 10 years after HSCT | Up to 24 months | Time from HSCT to first diagnosis of iron overload |
| Efficacy of curative treatments for iron overload in preventing hepatic complications | Up to 24 months | International non-proprietary names, dates and doses of treatments received for iron overload ≥ 10 years after HSCT |
Countries
France
Contacts
CONTACTMathilde Ruggiu, MD
CONTACTJérôme Lambert, MD PhD
Outcome results
None listed