MRD-BMT with Fludarabine-based RIC for Acquired AA for Anemia, Aplastic

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Children's Hospital of Philadelphia, Philadelphia, PA
Anemia, Aplastic+5 More
MRD-BMT with Fludarabine-based RIC for Acquired AA - Other
Eligibility
< 65
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a new type of bone marrow transplant may help individuals with aplastic anemia or inherited bone marrow failure syndromes.

See full description

Eligible Conditions

  • Anemia, Aplastic
  • Bone Marrow Failure Syndromes

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Anemia, Aplastic

Study Objectives

This trial is evaluating whether MRD-BMT with Fludarabine-based RIC for Acquired AA will improve 3 primary outcomes and 2 secondary outcomes in patients with Anemia, Aplastic. Measurement will happen over the course of Up to 100 days post transplant.

Year 1
Rate of disease free survival
Rate of graft failure
Rate of overall survival
Time to neutrophil engraftment
Day 100
Transplant-related mortality

Trial Safety

Safety Progress

1 of 3

Other trials for Anemia, Aplastic

Trial Design

3 Treatment Groups

Acquired Aplastic Anemia (AA)
1 of 3
Inherited Bone Marrow Failure Syndrome + Trilineage Aplasia
1 of 3
Inherited Bone Marrow Failure Syndrome no Trilineage Aplasia
1 of 3
Experimental Treatment

This trial requires 25 total participants across 3 different treatment groups

This trial involves 3 different treatments. MRD-BMT With Fludarabine-based RIC For Acquired AA is the primary treatment being studied. Participants will be divided into 3 treatment groups. There is no placebo group. The treatments being tested are in Phase < 1 and are in the first stage of evaluation with people.

Acquired Aplastic Anemia (AA)
Other
Patients with severe or very severe acquired aplastic anemia (AA). Patients will receive a matched related donor bone marrow transplant following reduced intensity conditioning (RIC) including thymoglobulin (ATG), fludarabine and dose-reduced cyclophosphamide.
Inherited Bone Marrow Failure Syndrome + Trilineage Aplasia
Other
Patients with inherited bone marrow failure (iBMF) syndromes with trilineage aplasia includes those with diagnoses of Fanconi Anemia, Dyskeratosis Congenita, and related conditions. Patients will receive a matched related donor bone marrow transplant following conditioning with fludarabine, cyclophosphamide, thymoglobulin.
Inherited Bone Marrow Failure Syndrome no Trilineage Aplasia
Other
Patients with inherited bone marrow failure (iBMF) syndromes without trilineage aplasia includes those with diagnoses of Severe Congenital Neutropenia, Diamond-Blackfan Anemia, and related conditions. Patients will receive a matched related donor bone marrow transplant following conditioning with thymoglobulin, busulfan and fludarabine.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 1 year post transplant
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 1 year post transplant for reporting.

Who is running the study

Principal Investigator
T. O.
Prof. Timothy Olson, Assistant Professor
Children's Hospital of Philadelphia

Closest Location

Children's Hospital of Philadelphia - Philadelphia, PA

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Patients 0-22 years with acquired aplastic anemia or a diagnosed inherited bone marrow failure syndrome, and a fully Human leukocyte antigen (HLA)-matched (10/10) related donor.
Ages 0-22 years at time of enrollment
Bone marrow biopsy demonstrating cellularity of <25% (at least 2 weeks from last dose of G-CSF), in addition to 2 of the following: absolute neutrophil count (ANC) <500/µL, platelets < 20,000/µL and absolute reticulocytes <40,000/µL
Negative evaluation for inherited bone marrow failure conditions and negative evaluation for dysplasia or cytogenetic abnormalities associated with myelodysplastic syndromes
Patients with concurrent paroxysmal nocturnal hemoglobinuria (PNH) clones are eligible, as long as they meet criteria for severe or very severe aplastic anemia as defined above
Fanconi Anemia
Dyskeratosis Congenita
Severe Congenital Neutropenia
Diamond-Blackfan Anemia
Congenital Dyserythropoietic/Sideroblastic Anemias

Patient Q&A Section

What is anemia, aplastic?

"This group summarizes the current state of knowledge regarding anemia and its impact on pediatric patients. In most respects, it provides clinicians with an unbiased overview of current evidence." - Anonymous Online Contributor

Unverified Answer

Can anemia, aplastic be cured?

"Anemic and aplastic anemias could be potentially cured with appropriate treatment and can be achieved at one third of the cost of the regular blood transfusion treatment. All patients receiving blood need to be fully informed about these possible positive results and the risks of the transfusion treatment." - Anonymous Online Contributor

Unverified Answer

How many people get anemia, aplastic a year in the United States?

"Around 40 million Americans have anemia, aplastic anemia each year; however it is difficult to obtain accurate prevalence estimates of both conditions since they are often not reported to health care providers." - Anonymous Online Contributor

Unverified Answer

What are the signs of anemia, aplastic?

"Mild symptoms of anemia or aplastic include loss of appetite and fatigue, and more severe manifestations of anemia or aplastic include a decrease in hemoglobin values, peripheral neuropathy, and anemia-related symptoms. If symptoms are not addressed and treated promptly, anemia or aplastic may evolve into a life-threatening condition." - Anonymous Online Contributor

Unverified Answer

What are common treatments for anemia, aplastic?

"Iron deficiency and low transferrin can be treated with oral iron supplementation but there is no specific treatment for anemia as a result of chronic blood loss." - Anonymous Online Contributor

Unverified Answer

What causes anemia, aplastic?

"Both hemoglobinopathies and other causes of anemia can cause anemia. Aplastic anemia can develop after bone marrow suppression, often due to cancer therapy. Hematopathologic examination demonstrates a reduction in circulating blood cells, as measured by their reduced surface area. These alterations are not readily reversible in most patients and usually require treatment with blood transfusions." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for anemia, aplastic?

"Clinical trials of therapy for anemia should include patients with low Hct and Apl < or = 15% at baseline. Clinical trials for aplastic patients should include patients with Apl > or = 10% or low Hct at baseline. Randomized trials may be of use for anemic and aplastic patients but, for anemic patients, they may have a greater risk of producing misleading results if Hct is the only outcome measure used." - Anonymous Online Contributor

Unverified Answer

How serious can anemia, aplastic be?

"The data obtained from HES and Hb can be used to anticipate hemoglobin level response to iron therapy; however, Hb and HES values cannot predict response to erythropoietin therapy for anemia and aplastic anemia." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in mrd-bmt with fludarabine-based ric for acquired aa for therapeutic use?

"The following are the present-day guidelines for the use of aFludarabine-based treatments for acute leukemia in the adult population. For those patients who do not achieve a complete remission, maintenance chemotherapy is recommended. For those patients who reach a complete remission, aFludarabine-based treatment lasting longer than 8 cycles is recommended." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets anemia, aplastic?

"The average age someone gets anemia, aplastic in a population without prior history of the disorder were 10-23 and 48-64+ years respectively. The average duration of the disorder in someone with aplastic anemia and iron deficiency anemia was 9-31+ and 11-55+ years respectively." - Anonymous Online Contributor

Unverified Answer

What is mrd-bmt with fludarabine-based ric for acquired aa?

"This new combination of fludarabine with the anthracycline-derived antibiotic mrd-bmt is safe and effective for Aa-associated disease. The long-term benefits of treatment should be investigated in a larger study." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving mrd-bmt with fludarabine-based ric for acquired aa?

"This is the first study to reveal significant clinical benefits of fd + brt/ric in a subset of individuals with AA who responded well to the aforementioned drugs and has suggested a novel drug-therapy combination in AA that warrants further investigation." - Anonymous Online Contributor

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