Myelodysplastic Syndrome, Myelofibrosis
Conditions
Keywords
Myelodysplastic syndrome, myelofibrosis, iron chelation therapy, haemodialysis
Brief summary
In transfusion-dependent myelodysplastic syndromes patients, regular blood transfusions lead to iron overload, which can cause organ damage, hormonal imbalances, and increased infection risk, ultimately impacting patient survival. Standard oral iron chelation therapies can be intolerable for some patients due to adverse effects. The MEX-CD1 device (class III) could potentially offer an alternative for these patients by reducing serum iron levels through a novel, extracorporeal approach. MEXIRON clinical investigation focuses on the use of MEX-CD1, a medical device designed for extracorporeal chelation therapy to reduce iron overload in patients suffering from transfusion-dependent myelodysplastic syndromes (MDS) and myelofibrosis. MEXIRON aims to evaluate the device's use feasibility, safety, and effectiveness in reducing iron levels. Transfusions needs, patient experience and quality of life are also assessed. Each enrolled patients will undergo three low-volume continuous veno-venous haemodialysis cycles within one week. Following the three- haemodialysis cycles, patients will be monitored through on-site follow-up visits at 7 days, 28 days, and 90 days post-treatment to assess long-term effects.
Interventions
DEVICELow-volume continuous veno-venous haemodialysis with MEX-CD1 use
MEX-CD1 is a hyper-chelating colloidal solution that can be added to the dialysate to be used in low-volume continuous veno-venous hemodialysis. Intervention includes 3 low-volume continuous veno-venous hemodialysis for a duration of 3h20 each. For non-hospitalized patients, the treatment is performed on an outpatient basis.
Sponsors
Hospices Civils de Lyon
MEXBrain 13 avenue Albert Einstein 69100 Villeurbanne
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Feasibility, prospective, monocentric, pilot clinical investigation
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patient followed for myelodysplastic syndrome or myelofibrosis. * Patient with platelet count ≥50 giga/L at inclusion. * Patients with severe anemia and hemoglobin \<70 g/L at baseline. * Patient with intolerance or contraindication to oral or subcutaneous chelation therapy. * Ferritinemia \>1000 µg/L or hepatic iron concentration ≥7 mg/g or cardiac T2\* \<20 ms at inclusion. * Patient able to understand (French-speaking) and comply with protocol, having signed informed consent.
Exclusion criteria
* Patients with primary hemochromatosis (transferrin saturation coefficient CS-Tf \> 45%). * Patients with a contraindication to the use of MEX-CD1: weight \< 30 kg, iron deficiency. * Patients with a known allergy or contraindication to heparin or citrate. * Patients undergoing azacitidine or other chemotherapy (or considered as such) for myelodysplastic syndrome or myelofibrosis. * Patient who received treatment with luspatercept or erythropoietin (EPO) during the month prior to inclusion. * Patients with indications for allogeneic bone marrow transplantation. * Patients with a known allergy to shellfish (MEX-CD1 contains chitosan of animal origin) or to one of the other components of MEX-CD1. * Patients with a peripheral vascular access that is difficult to access or that needs to be preserved. * Patients participating in other interventional research that could interfere with the results of the study. * Patients under legal protection or unable to express their consent. * Patients under psychiatric care. * Patient deprived of liberty by judicial or administrative decision. * Pregnant or breast-feeding women.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Performance of MEX-CD1 on total iron chelation during a session epuration. | Day 4 | Quantity of iron values measured in the effluent bag (Hf) during each low-volume continuous veno-venous hemodialysis cycle |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Performance of MEX-CD1 on iron profile. | Day 4 | Amount of iron measured on the effluent line (H1, H2, Hf) during each Extracorporeal Purification session |
| Safety of MEX-CD1 use | week 16 | Assessment of adverse events during low-volume continuous veno-venous hemodialysis cycles and all follow up period. |
| Impact on blood products patient's needs. | week 16 | Assessment on patient's red blood cells administration from the start of the first MEX-CD1 treatment until the follow-up ending. |
| Feasibility of MEX-CD1 use | Day 4 | Assessment of patient's proportion who will have performed i) the entire first low-volume continuous veno-venous hemodialysis cycle and ii) the whole weakly 3 consecutive cycles at the end of the third low-volume continuous veno-venous hemodialysis cycle. |
| Patient's reported tolerability of low-volume continuous veno-venous hemodialysis cycle. | Day 4 | Patients tolerability assessment by Visual Analog Scales (VAS) on painful and asthenia feelings at the end of each low-volume continuous veno-venous hemodialysis cycle. |
| Patient's reported quality of life. | Week 16 | Quality of life assessment by the Medical Outcome Study Short Form 12 from the inclusion visit until end of follow-up. |
Countries
France
Contacts
CONTACTAuguste DARGENT, MD, PhD
CONTACTBernard ALLAOUCHICHE, MD
Outcome results
None listed