Clinical trial NCT03659084: Study of the Outcome of Patients With Acute Myeloblastic Leukemia and Myelodysplastic Syndrome Receiving Iron Chelation Therapy After Allogeneic Hematopoietic Stem Cell Transplantation
Official title: Multicenter Prospective Observational Study of the Outcome of Patients With Acute Myeloblastic Leukemia (AML) and Myelodysplastic Syndrome (MDS) Receiving Iron Chelation Therapy (Exjade) After Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Myeloid Leukemia
Myelodysplastic Syndromes
Keywords: Acute myeloid leukaemia
Myelodysplastic syndrome
Iron Chelation Therapy (Exjade)
allogeneic Hematopoietic Stem Cell transplantation (Allo-HSCT)
Lead sponsor: Hospices Civils de Lyon
Sponsor class: OTHER
First posted: 2018-09-06
Enrollment: 150
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with acute leukemia (AML) or Myelodysplastic Syndrome (MDS) and iron overload with ferritinemia ≥ 1000 μg / l, 6 months after CSH allograft.

Brief summary: Iron chelation, mostly associated with multiple red blood cell transfusion, is relatively common in patients with hematological malignancies receiving allo-HSCT.

This multicenter prospective observational study is designed to establish the impact of iron chelation on relapse after allo-HSCT in patients with acute myeloid leukemia and myelodysplastic syndrome. The investigators will compare the results obtained in the prospective study to those observed in a historical retrospective cohort of paired patients who did not receive chelation. Given our clinical experience and literature results, the investigators will evaluate the Exjade chelator. Although not demonstrated, the presence of mutations of the HFE gene could play an indirect role on leukemogenesis by promoting overload. It is therefore important to evaluate the status in this patient population.


Eligibility criteria: Inclusion Criteria:

* adults older than 18 years old
* Patients with AML or MDS in complete remission receiving CSH transplantation from a related or unrelated donor and after myeloablative or non-myeloablative conditioning.
* Patients with iron overload defined by at least one ferritinemia\> 1000 μg / L in the 6th month after CSH allograft
* Creatinine less than 1.5 x ULN; ALAT and ASAT \<2 x ULN
* Patients giving their informed consent (prior to performing any study procedure)

Exclusion Criteria:

* Hypersensitivity to the Exjade
* Association with another iron chelator
* Proteinuria\> 1g / 24h
* Acute and chronic hepatitis (B and C viruses); HIV
* Extended corrected QT
* History of ocular toxicity related to iron chelation treatment
* Gastrointestinal Abnormal Absorption of Oral Medications
* Pregnancy and lactation

Primary outcomes:
measure: Impact of iron chelation on relapse-free survival rate
timeFrame: At 2 years
Secondary outcomes:
measure: Comparison of relapse-free survival after allograft of chelated patients to allografted patients not receiving chelation.
timeFrame: At 2-year
measure: Cumulative incidence of GVHD
timeFrame: 3 months, 1 and 2 years
measure: Rate of infection
timeFrame: Through study completion, an average of 4 years
measure: Hematological toxicity during administration of Exjade
timeFrame: Through study completion, an average of 4 years
measure: Non-hematological toxicity during administration of Exjade
timeFrame: Through study completion, an average of 4 years
measure: Non-hematological toxicity during administration of Exjade
timeFrame: Through study completion, an average of 4 years
Other outcomes:
Locations:
city: Pierre-Bénite
country: France
facility: Centre Hospitalier Lyon Sud
Study officials:
affiliation: Hospices Civils de Lyon
name: Mauricette MICHALLET, MD
role: PRINCIPAL_INVESTIGATOR
References: