Treatment for AML, Childhood

Phase-Based Estimates
1
Effectiveness
1
Safety
Texas Children's Hospital/Baylor College of Medicine, Houston, TX
Eligibility
< 65
All Sexes
Eligible conditions
AML, Childhood

Study Summary

This study is evaluating whether a combination of two drugs may help treat children with leukemia.

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Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Treatment will improve 2 primary outcomes in patients with AML, Childhood. Measurement will happen over the course of 4 weeks.

4 weeks
Dose Toxicity
Remission

Trial Safety

Safety Estimate

1 of 3

Trial Design

1 Treatment Groups

Control

This trial requires 26 total participants across 1 different treatment groups

This trial involves a single treatment. Treatment is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

ControlNo treatment in the control group

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 4 weeks
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 4 weeks for reporting.

Closest Location

Texas Children's Hospital/Baylor College of Medicine - Houston, TX

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. You must have received 1 prior treatment for AML, Childhood. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
1st or greater relapse, OR Failed to go into remission (i.e. refractory) after first or greater relapse, OR Failed to go into remission from original diagnosis after two or more induction attempts.
Patients with DNA fragility syndromes (such as Fanconi anemia, Bloom syndrome) are excluded.
Age Patients must be ≥ 1 and ≤30 years of age.
Diagnosis
Relapsed or refractory AML with ≥5% blasts (by morphology) in the bone marrow.
Relapsed or refractory AML with ≤ 5% blasts (by morphology) and MRD positive disease (M1/MRD+): Two serial marrows demonstrating stable or rising MRD ≥ 0.1 % (i.e. not declining). MRD will be determined by multiparameter flow cytometry using AML-associated phenotype markers, or real-time quantitative PCR for AML-associated genetic lesions
Patients may have CNS 1 or 2 or other sites of extramedullary disease. No cranial irradiation is allowed during the protocol therapy.
Patients with secondary AML are eligible.
Patients with Down Syndrome will be eligible and will be included as an observation cohort
Performance Level Karnofsky > 50% for patients > 16 years of age and Lansky > 50% for patients ≤ 16 years of age.

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What causes aml, childhood?

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Aml, childhood may be a significant factor that predisposes children, particularly those with a first-degree relative with the condition, to developing later life-threatening, non-metastatic cancer, especially childhood leukemia and, less commonly, acute lymphoblastic leukemia. (Bibliography pgs. 5, 7).

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How many people get aml, childhood a year in the United States?

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About 11.8 million children live in the United States, so it is estimated that an average of one out of five children has been diagnosed with aml, childhood in the past year. The estimates for aml, childhood vary because of country, so it is recommended to use the National Epidemiologic Survey on Alcohol and Related Conditions estimates, adjusted by age, to calculate the number of people with aml, childhood. The National Healthcare Statistics System provides other estimates.

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What is aml, childhood?

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Aml, childhood, used to refer to multiple myeloma in children, refers to an accumulation of plasma cells in the bone marrow. It can form in any age group, but it is generally diagnosed at an older age and in a male. A diagnosis of aml is not associated with any particular medical problems, and typically does not kill a child.

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What are the signs of aml, childhood?

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Symptoms of amL are a spectrum and vary in prevalence and severity. The most common symptoms are muscle weakness and fatigue. Seizures, ataxia, and apathy are less common symptoms.

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Can aml, childhood be cured?

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AML, childhood can not be cured but can be improved significantly through structured comprehensive approaches. Early detection and comprehensive therapy can help reduce symptom level and improve quality of life in AML, childhood.

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What are common treatments for aml, childhood?

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As of early 2012, the evidence that indicated one treatment as “superior” to another had not been published. As of late 2012, the evidence indicates that there were no known significant clinical differences between one form of treatment and another when used for aml, childhood. It is likely safe to use the methods and/or treatments cited in the present article if used correctly, according to the patient's instructions.

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Is treatment safe for people?

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Recent findings of this study suggest that the benefits of treatment outweigh the risks to people with hyperactive behaviour and co-occurring ADHD on an ADHD-only group. Recent findings do not extend beyond the ADHD-only group and do not show a benefit to the other groups. Overall, the study does not give a strong argument against treatment, and instead suggests the need for more research.

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Have there been other clinical trials involving treatment?

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The only documented clinical trial involving anti-inflammatory aml for the treatment of SSPE was performed in 1978. A similar randomized, prospective, double-blinded clinical trial aimed to study the immunomodulatory role of aml in patients with SSPE was started in 1999. The study is currently ongoing; at this time the results have not been published. The study design did not include a placebo treatment arm and no randomized control trial has yet been performed. At this time, the exact clinical protocol used was not known. Further evaluation of aml in SSPE is now mandatory, and its possible role should be examined in further clinical trials. The clinical trial of aml for the treatment of SSPE is currently underway.

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Does aml, childhood run in families?

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In the current study we did not see a trend between having a sibling with childhood aml and having either a positive or negative family history for the disease. As such we did not find any compelling evidence that the pathobiology of childhood aml, run in families. Future studies will need to address the question of whether other factors are involved in the etiology of childhood aml and whether the findings in this study can be generalized to other populations.

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How serious can aml, childhood be?

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Results from a recent clinical trial indicate that ACS-CFS/ME in childhood and adolescence can have a profound effect on the health of the individuals and their families, thereby necessitating a lifelong need for treatment. Further, ACS-CFS/ME in childhood is associated with reduced quality of life when reported by parents. Results from a recent clinical trial suggest that children and adolescence are particularly vulnerable to experiencing the consequences of illness that can be associated with chronic pain and fatigue, and the impact of ACS-CFS/ME on the quality of life and health of the sufferer and their families may be particularly dire.

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Is treatment typically used in combination with any other treatments?

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The most frequently used combination of therapies differed by patient characteristics (young age and/or first diagnosis during treatment versus later in treatment for adults and/or relapse) and by diagnosis (leukemia, CNS disease, rhabdomyosarcoma and/or Ewing disease among children, and retinoblastoma or hepatoblastoma among adults). Understanding these differences provides important insight into treatment regimens for specific cancers.

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What are the common side effects of treatment?

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It is important for patients and health professionals to understand the common side effects of HCCA, which should be discussed with patients before any type of therapy is started or with new patients who have been treated before. HCCA carries the risk of cardiotoxicity, which is a concern in patients with heart disease or who are taking cardiac risk factors (hypertension, smoking, hyperlipidemia, etc.), but will decrease with adequate therapy. Severe thrombocytopenia or aplastic anemia has been seen rarely. The use of oral contraceptives, especially after HCCA therapy, increases the risk for venous thrombosis.

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