Symptoms and General Pathology

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Etoposide

Cyclophosphamide

Lorazepam

Methotrexate

Etoposide phosphate

Busulfan

Total Body Irradiation (TBI) 1200 cGy will be given on days -8,-7,-6 and -5 in eight sessions, delivering 150cGy in each session.

Etoposide 1000 mg/m2 as a 24 hour continuous infusion started on day -4.

Cyclophosphamide 60 mg/kg/day IV given over 1 hour daily on days -3, -2.

Myeloablative Chemotherapy Regimen for Lymphoid Malignancies or Cord Blood Unit Recipients

Busulfan administration:

* For children \>/= 4 years of age, Busulfan 0.8 mg/kg/dose will be given every 6 hours over days -8,-7, -6, and -5 for a total of 16 doses.
* For children \< 4 years of age, Busulfan 1 mg/kg/dose will be given every 6 hours over days -8, -7, -6, -5 for a total of 16 doses.
* Pharmacokinetic analysis will guide dose modifications targeted to receive an average AUC of 800-1200 microMols\*min for the 16 doses.

Lorazepam (0.05 mg/kg) IV will be administered one half hour before the initial dose of Busulfan is given and every 6 hours through day -4.

Etoposide 1000 mg/m2 as a 24 hour continuous infusion started on day -4.

Cyclophosphamide 60 mg/kg/day IV given over 1 hour daily on days -3 and -2.

Myeloablative Chemotherapy Regimen for Non-Cord Blood Unit Recipients with Myeloid Malignancies

Hematopoietic progenitor cells (HPCs) will be infused on day 0. Source of cells may be bone marrow, peripheral blood cells, or cord blood units, from matched related or unrelated donors.

Hematopoietic Progenitor Cell Transplantation (HPCT)

Patients with prior CNS disease over the age of 1 year will be treated with 600 cGy of cranial irradiation in addition to 1200 cGy of TBI.

Patients diagnosed with ALL with CNS disease (at the time of diagnosis or relapse) \< 1 year of age will receive CNS treatment as Intrathecal Methotrexate as follows:

* Infants ≤ 1 year of age at the time of Intrathecal Therapy will receive a dosing of 7.5 mg once a month for 6 months after transplant beginning at day +30 with an adequate white count
* Children 1-2 years of age at the time of Intrathecal Therapy will receive 8 mg once a month for 6 months after transplant beginning at day +30 with an adequate white count

CNS radiation treatment for ALL with prior CNS disease patients

Morris Kletzel, MD

Terriss Conterato

The purpose of this study is to show that myeloablative hematopoietic progenitor cell transplantation (HPCT) continues to offer acceptable disease-free survival for select patients requiring HPCT.

Myeloablative hematopoietic progenitor cell transplantation (HPCT) remains the standard of care for patients requiring HPCT. The purpose of this study is to evaluate the morbidity and mortality of myeloablative HPCT at Children's Memorial Hospital. It will also look to determine the toxicity of a single conditioning regimen consisting of total body irradiation (TBI), etoposide (VP-16), and Cyclophosphamide for patients with transplant eligible lymphoid malignant conditions or with transplant eligible myeloid malignant conditions who are receiving cord blood units, or to determine the toxicity of a single conditioning regimen consisting of Busulfan and Cyclophosphamide for patients with transplant eligible myeloid malignant conditions who are not receiving cord blood units.

Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies

Ann & Robert H Lurie Children's Hospital of Chicago

Nutritional and Metabolic Diseases

Anemia

Anemia, Aplastic

Iron Overload

Bone Marrow Failure Disorders

Pancytopenia

Metabolic Diseases

Iron Metabolism Disorders

Precancerous Conditions

Aplastic Anemia

Micronutrients

Iron

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.

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

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

Novartis

Peking Union Medical College Hospital

Department of Hematology

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies	NCT00619879	Leukemia, Myelo... Leukemia, Lymph... Leukemia, Myelo... Myeloproliferat... Lymphoma, Malig...	Myeloablative C... Myeloablative C... Hematopoietic P... CNS radiation t...	- 21 Years	Ann & Robert H Lurie Children's Hospital of Chicago
Study of MRI Monitoring in Patients With Aplastic Anemia and Low or Int-1 Risk of MDS Complicated With Iron Overload	NCT02833493	Aplastic Anemia Dysmyelopoietic... Iron Overload		18 Years - 80 Years	Peking Union Medical College Hospital

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