Aplastic Anemia Clinical Trial
Official title:
Observational Study of MRI Monitoring in Patients With Aplastic Anemia (AA) and Low or Int-1 Risk of Myelodysplastic Syndromes(MDS) Complicated With Iron Overload
The investigators aim to give an overview of Iron overload(IOL) of patients with AA and low and int-1 risk MDS and their sequelae under different chelation treatment. And the investigators also aim to evaluate the relationship of LIC and T2*/R2*.
Long-term transfusion therapy, a supporting treatment for patients with intractable chronic
anemia is currently the most frequent cause of secondary iron overload. Both Aplastic anemia
(AA) and low risk (low and intermediate-1 risk) myelodysplastic syndromes (MDS) are
classified into bone marrow failure syndromes (BMFs) as they have a lot of characters in
common. Iron overload (IOL) can then become a significant problem in regularly transfused
patients, leading to organ damage, particularly in the liver and heart. Iron overload also
has a suppressive effect on erythroid progenitors and may increase transfusion requirements.
In those cases, iron chelation therapy may help to improve their quality of life and prolong
their survival.
Because of the importance of iron chelation in patients with AA and low and
intermediate-1(int-1) risk MDS complicated with iron overload, it is necessary to monitor
their iron overload status to find the suitable patients to be chelated and follow up the
effectiveness of therapy. Using quantitative Magnetic resonance imaging (MRI) T2* to detect
the iron deposit of different organs has been introduce to China since 5 years ago. Compared
to the traditional methods for evaluating iron overload like clinical manifestations, serum
ferritin (SF) level, transferrin saturation (TS), CT and echocardiography (UCG) etc., which
are widely used so far in China, MRI T2* provides an more accurate, convenient and
affordable non-invasive way of monitoring iron overload. More important, it is very reliable
to monitor the improvement of iron chelation therapy since the variation of MRI detection
between different detections is very low. Few reports have been focused on IOL of MDS and AA
in China so far.
Measurement of liver iron concentration (LIC) by MRI yields similar results to those coming
from liver biopsy analysis, and is a validated tool for detection of iron overload. Data has
been published from a multi-center trial evaluating the efficacy and safety of deferasirox
(DFX) in low and intermediate-1 risk MDS patients with transfusion-dependent IOL and showed
DFX yields sufficient reduction of excess iron indicated by serum ferritin levels and most
importantly by liver MRI. But the median duration of DFX treatment is only 354 days and no
data of Chinese patients was included. Most of the studies for MDS lack data of long term
follow-up and there is scarcely any data on AA so far.
In China, more and more patients with iron overload can afford adequate iron chelation
therapies, although there are still some patients who cannot afford at all or can only be
chelated irregularly. And some patients can only accept deferoxamine instead of deferasirox
because of the medical insurance policies. It is important to include patients with
different situations and monitor their iron change in their major organs based on different
chelation level.
In this study, it is anticipated to evaluate prospectively 80 patients with AA and low or
int-1 risk MDS with IOL, by the traditional methods and MRI T2*. Clinical parameters and
T2*values will be monitored every 12 months for 3 years. Other parameters like clinical
follow-ups ( rate of infection, liver disease, cardiac disorders, endocrine function and
other co-morbidities associated with MDS/AAs, etc.), SF, liver and kidney function, UCG
tests will be monitored as well at the interval of every 6 months. At the end of the study,
patients will be classified as well chelated groups (defined as those received deferasirox
20mg/kg or deferoxamine 40 mg/kg for more than 255 days/year) or poor chelated groups
(defined as those received iron chelation therapy dose less than above) and compared the
differences of their outcome and change of iron status. The investigators aim to give an
overview of IOL of patients with AA and low and int-1 risk MDS and their sequelae under
different chelation treatment. And the investigators also aim to evaluate the relationship
of LIC and T2*/R2*. It is the first and longest prospective clinical trial on AA and low
risk MDS and will give us a better understanding of the value of proper chelation treatment
for the organ function.
;
Observational Model: Case-Only, Time Perspective: Prospective
Status | Clinical Trial | Phase | |
---|---|---|---|
Active, not recruiting |
NCT03025698 -
A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia
|
Phase 2 | |
Completed |
NCT00987480 -
Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
|
Phase 2 | |
Completed |
NCT00767650 -
Neuropsychological Effects of Immunosuppressive Treatment in Subjects With Aplastic Anemia
|
N/A | |
Completed |
NCT02833805 -
NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia
|
Phase 2 | |
Recruiting |
NCT02028416 -
Comparison of Two Different Doses of Rabbit ATG-Fresenius With Cyclosporin in the Treatment of Acquired Aplastic Anaemia
|
N/A | |
Completed |
NCT00004474 -
Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia
|
Phase 3 | |
Recruiting |
NCT05031897 -
Reduced-Intensity Conditioning for the Prevention of Treatment-Related Mortality in Patients Who Undergo a Hematopoietic Stem Cell Transplant
|
Phase 2 | |
Completed |
NCT04439006 -
Ibrutinib for the Treatment of COVID-19 in Patients Requiring Hospitalization
|
Phase 1 | |
Not yet recruiting |
NCT05996393 -
CsA+ATG+AVA vs. CsA+AVA for the Treatment of Newly-diagnosed SAA in the Elderly
|
Phase 4 | |
Completed |
NCT02462252 -
Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome
|
Phase 2 | |
Completed |
NCT01272817 -
Nonmyeloablative Allogeneic Transplant
|
N/A | |
Completed |
NCT00513175 -
Non-Myeloablative Allogeneic Stem Cell Transplantation With Matched Unrelated Donors for Treatment of Hematologic Malignancies, Renal Cell Carcinoma, and Aplastic Anemia
|
N/A | |
Completed |
NCT00001398 -
Stem Cell Factor Medication for Aplastic Anemia
|
Phase 1 | |
Recruiting |
NCT01861093 -
Safety Study of Cord Blood Units for Stem Cell Transplants
|
Phase 2 | |
Not yet recruiting |
NCT05018936 -
Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia
|
Phase 2/Phase 3 | |
Completed |
NCT00065260 -
Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia
|
Phase 2 | |
Recruiting |
NCT02007811 -
Open-label Clinical Trial to Investigate the Safety and Tolerability of Allogeneic B-cell Concentrates for Immune Reconstitution After Allogeneic Stem Cell Transplantation Measured as Response to a Antedated Single Vaccination
|
Phase 1/Phase 2 | |
Recruiting |
NCT01758042 -
Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders
|
N/A | |
Terminated |
NCT01500161 -
Pooled Unrelated Donor Umbilical Cord Blood Transplant For Hematologic Malignancy Needing Allogeneic Stem Cell Transplant Without Related HLA-Match
|
Phase 2 | |
Recruiting |
NCT00882323 -
Reduced Toxicity Fludarabine (Flu) + Cyclophosphamide (CPM) + Rabbit Antithymocyte Globulin (rATG) Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia (SAA)
|
Phase 2 |