Non Interventional Study on Iron Toxicity After First Allo-transplant in MDS/CMML

NCT06267898 | Completed

• 1 other identifier: EBMT- 842205547
• Study Type: observational
• Target: 222
• Locations: 3 countries
• Sites: 6

Brief Summary

Stem cell transplantation and blood product transfusions are standard of care for Myelodysplastic Syndromes (MDS). Several studies have shown changes in serum ferritin and non-transferrin-bound iron (NTBI) in patients undergoing stem cell transplantation. A large proportion of MDS patients are at risk for organ damage from tissue siderosis, due to the development of iron overload. Toxic effects of iron may play an important role in the complications associated with HSCT. Iron chelation therapy may reduce the acute and chronic treatment-related toxicity by removing excess of iron, iron radicals and reactive oxygen species (ROS). There is little information about the efficacy and safety of iron chelation in MDS patients. This audit wants to evaluate the effect of iron toxicity on treatment-related mortality in untreated, adult MDS or CMML patients during and after treatment with myeloablative conditioning (MAC) and reduced intensity conditioning (RIC) allo-HSCT, by prospectively collecting data from 200 MDS or CMML patients from 2009 onwards.

Conditions

MDS; CMML

Keywords

Reduced Intensity conditioning; Myeloablative conditioning; Allogeneic stem cell transplantation; iron toxicity; Non-interventional; Observational study; MDS; CMML

Outcome Measures

Primary Outcomes (1)

• non-relapse mortality (treatment related mortality).

  2 years after HSCT

Secondary Outcomes (4)

• treatment-related toxic effects

  2 years after HSCT

• relapse rate

  2 years after HSCT

• event-free survival

  2 years after HSCT

• overall survival

  2 years after HSCT

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

eligible patient from EBMT centres

You may qualify if:

• Patients with MDS or CMML, according to FAB-criteria (RA, RARS, RAEB, RAEBt, CMML) or according to WHO-criteria (RA, RARS, RAEB-1, RAEB-2, RCMD, RCMD-RS, with isolated del(5q), MDS OR CMML-U)
• Patients with transformed MDS or CMML to AML at time of transplant
• Patients received myeloablative or RIC allo-HSCT as primary treatment
• Diagnosis at time of transplant
• Informed Consent and of legal age at the time of obtaining informed consent (18+)
• ECOG performance status 0-2

You may not qualify if:

• Patients with previous intensive antileukemic therapy (intensive chemotherapy and/or HSCT); previous treatment with immunomodulatory drugs (thalidomide, or lenalidomide) or hypomethylating agents (Vidaza, decitabine) is allowed
• Patients with juvenile chronic myelomonocytic leukemia (jMML)
• Patients with secondary or therapy-related AML and MDS or CMML after treatment with immunosuppressive or cytotoxic treatment for a nonmyeloid malignancy
• Patients who received auto-HSCT
• Candidates for cord blood HSCT or syngeneic HSCT
• Inadequate renal function (ECC \<60 ml/min and/or creatinine \>2.5 times upper limit of normal value)
• Inadequate hepatic function (transaminases \>2.5 times upper limit of normal value)
• History of seizures
• Pregnancy and women of child-bearing potential and not using adequate contraceptives
• Uncontrolled hypertension

Sponsors & Collaborators

• European Society for Blood and Marrow Transplantation: lead
• Novartis: collaborator

Study Sites (6)

Antwerp University Hospital (UZA)

Antwerp, 2650, Belgium

Location

University Hospital Gasthuisberg

Leuven, 3000, Belgium

Location

University of Liege

Liège, 4000, Belgium

Location

Charles University Hospital

Pilsen, 304 60, Czechia

Location

University Hospital

Essen, 45122, Germany

Location

University Hospital Leipzig

Leipzig, 04103, Germany

Location

Study Officials

• T. de Witte, MD

  Radboud University Medical Center

  PRINCIPAL INVESTIGATOR

• E. Cremers

  VUMC - VU University Medical Centre Amsterdam

  PRINCIPAL INVESTIGATOR

• N. Kröger, MD

  Universitätsklinikum Hamburg-Eppendorf

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Target Duration: 2 Years
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 14, 2019
• First Posted: February 20, 2024
• Study Start: November 1, 2009
• Primary Completion: January 29, 2015
• Study Completion: May 21, 2015
• Last Updated: February 20, 2024

Record last verified: 2024-02

Locations

BE (3); DE (2); CZ (1)