Study of MRI Monitoring in Patients With Aplastic Anemia and Low or Int-1 Risk of MDS Complicated With Iron Overload

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Brief Title

Study of MRI Monitoring in Patients With Aplastic Anemia and Low or Int-1 Risk of MDS Complicated With Iron Overload

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

Brief Summary

      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*.
    

Detailed Description

      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.
    


Study Type

Observational


Primary Outcome

MRI T2*/R2* for liver and heart


Condition

Aplastic Anemia



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

80

Start Date

September 2016

Completion Date

October 2019

Primary Completion Date

September 2019

Eligibility Criteria

        Inclusion Criteria:

          1. Male or female of 18-80 year old.

          2. With blood transfusion history or RBC-transfusion-dependence.

          3. BM smear and biopsy plus chromosome analysis within 3 months before signing ICF,
             otherwise done during screening.

          4. Excluding other diseases which might cause hematological abnormalities.

          5. Serum ferritin level≥500ng/ml with no sign of active infection or malignant disease.

          6. Treatment with underlying disease is permitted for non-hematological and hematological
             conditions.

          7. Previous iron chelation therapy like deferasirox or deferoxamine is permitted.

          8. ECOG performance score ≤2

          9. All subjects must: agree not to donate blood or be counseled about pregnancy
             precautions and risks of fetal exposure.

         10. Written informed consent.

        Exclusion Criteria:

          1. Patients who are under 18-year-old or over 80-year-old.

          2. Prior history of other cancer unless cancer-free for ≥5 years.

             Subjects with the following history/concurrent conditions may enroll at any time:

             Basal cell carcinoma of the skin Squamous cell carcinoma of the skin Prostate cancer
             stage-1

          3. Proved HIV-1 infection

          4. Active HBV or active HCV infection.

          5. Pregnant or lactating

          6. Patients unwilling to or unable to comply with the protocol.
      

Gender

All

Ages

18 Years - 80 Years

Accepts Healthy Volunteers

No

Contacts

, , 



Administrative Informations


NCT ID

NCT02833493

Organization ID

CICL670AAU05


Responsible Party

Principal Investigator

Study Sponsor

Peking Union Medical College Hospital

Collaborators

 Novartis

Study Sponsor

, , 


Verification Date

July 2016