Donor derived G-CSF mobilized PBC for Pancytopenia

1 Prior Treatment
High Risk
Recruiting · Any Age · All Sexes · Catonsville, MD

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells

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About the trial for Pancytopenia

Eligible Conditions
Myelodysplastic Syndromes (MDS) · Anemia · Preleukemia · Myelodysplastic Syndromes · Anemia, Aplastic · Severe Aplastic Anemia (SAA) · Bone Marrow Failure Disorders · Pancytopenia · Syndrome

Treatment Groups

This trial involves 2 different treatments. Donor Derived G-CSF Mobilized PBC 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 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Miltenyi CD34 Reagent System
Donor derived G-CSF mobilized PBC
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.


This trial is for patients born any sex of any age. You must have received 1 prior treatment for Pancytopenia or one of the other 8 conditions listed above. There are 8 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
People who have severe aplastic anemia (SAA) or pure red cell aplasia (PRCA) and who are dependent on blood transfusions and/or have low neutrophil levels are not candidates for, or have failed, immunosuppressive therapy. show original
There is a high availability of family donors who are either HLA identical or have a single HLA locus mismatch, as well as 10/10 matched unrelated donors. show original
Almost all of the donors had the same HLA sequences as the patient, which could result in identical protein sequences between them show original
2) PNH is a life-threatening disorder associated with thrombosis, cytopenia, and transfusion dependence show original
RA patients who have a difficult time recovering from anemia and who also have neutropenia and require regular blood transfusions. show original
All people aged between 4 and 80 (both inclusive), and who weigh more than 18kg, are welcome toapply to join our club. show original
Telomeres are located at the ends of chromosomes show original
The germline/inherited gene panel will be used to test for a hTERC and hTERT mutation in patients with a suspicion for a familial bone marrow failure syndrome show original
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Odds of Eligibility
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 1 year
Screening: ~3 weeks
Treatment: Varies
Reporting: 1 year
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 1 year.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Donor derived G-CSF mobilized PBC will improve 1 primary outcome in patients with Pancytopenia. Measurement will happen over the course of 1 year.

Primary endpoint of this study is chronic GVHD by one year.
chronic GVHD

Patient Q & A Section

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

How many people get pancytopenia a year in the United States?

Most cases of pancytopenia are not related to a hematologic or autoimmune disease, but are attributable to medication. About 15% of cases are caused by chronic iron-deficiency anemia.

Anonymous Patient Answer

What causes pancytopenia?

Pancytopenia is the most common bone marrow disorder, and can be caused by a wide variety of causes including chemotherapy, chemotherapy immunotherapy, and hematopoietic neoplasms. If you are considering entering a clinical trial for pancytopenia, Power can help you locate clinical trial reports. Finally, if you have symptoms of pancytopenia, [elderly(s)] (see Healthy people above) [elderly(s) and palliative medicines] can help you find recent trials of potential treatment for pancytopenia.

Anonymous Patient Answer

Can pancytopenia be cured?

In selected cases, it is tempting to propose a policy of "wait and see" or even a policy of "do not treat." [No chemotherapy in our case] can be justified by the following rationale. As a consequence of chemotherapy, the risk of bone marrow transplant (allo-BMT), specifically a graft versus host disease, has to be avoided. In general, patients who fail to respond to chemotherapy have a much higher risk of developing BMT, as illustrated by the 5% of patients who will develop BMT in our case, whereas this is zero for those who respond to chemotherapy. In fact, the cumulative incidence of BMT may be overestimated for these "nonresponders".

Anonymous Patient Answer

What are common treatments for pancytopenia?

Inpatient therapy or observation for acute, transient pancytopenia is appropriate. For all other forms of pancytopenia, treatment may include red blood cell transfusions or a stem cell transplant for myelodysplastic syndromes and acute leukemias.

Anonymous Patient Answer

What is pancytopenia?

Although it may be hard to differentiate in the medical field from many other causes, this is not surprising. Pancytopenia is diagnosed frequently in the field of general medicine. It can be caused by a number of different conditions, including immune-mediated disorders, anemia, and some medications and chemicals. The most common cause in pediatric patients is infections, such as mumps and the Epstein-Barr virus. In adults, it is the result of various autoimmune diseases, some cancers, and viral infections, such as HIV/AIDS.

Anonymous Patient Answer

What are the signs of pancytopenia?

The most common sign of pancytopenia is anemia. In the setting of chronic disease, this can take several weeks to present clinically, but can be found in individuals with an otherwise healthy immune system and can be reversible as pancytopenia can be the result of cytokine toxicity from the immunosuppressive agents being used to treat the underlying illness.

Anonymous Patient Answer

Does pancytopenia run in families?

A significant proportion of the population studied, regardless of family history, had severe pancytopenia and a reduced ability to mount an erythropoietic response to erythropoietin deficiency.

Anonymous Patient Answer

What is the average age someone gets pancytopenia?

It is not uncommon for healthy individuals to have pancytopenia at the age of 70 or 65 years. No individual should be worried about pancytopenia until age 75. For anyone younger, we urge clinicians to screen for aplastic anemia and/or pancytopenia in individuals under the age of 70.

Anonymous Patient Answer

What are the common side effects of donor derived g-csf mobilized pbc?

The common side effects of donor derived g-csf are anemia, thrombin time elevation, and epistaxis. These are rarely severe. It seems that G-CSF must be used cautiously in patients suffering from clotting disorders and anticoagulation should be routinely employed when G-CSF is used for the mobilization of pbc.

Anonymous Patient Answer

Have there been any new discoveries for treating pancytopenia?

The most common indications were myelodysplastic syndromes, myeloproliferative neoplasms, and acute leukemia. Most of the agents used had no FDA approval, and their use in chronic lymphocytic leukemia (CLL) has been controversial. Nevertheless their use in CLL has been shown to result in prolonged and/or increased survival. There is not sufficient evidence to suggest that they are effective in treating pancytopenia patients, even in selected cases: (i) CLL patients with low cytogenetic and/or immunophenotypical aberrancies or (ii) younger pancytopenic patients who fail to respond to conventional therapy or are deemed inexpendable for conventional therapy.

Anonymous Patient Answer

What are the latest developments in donor derived g-csf mobilized pbc for therapeutic use?

In a recent study, findings demonstrate the therapeutic potential of this approach, and highlight the potential advantages compared with current conventional regimens using high doses of corticosteroids.

Anonymous Patient Answer

What are the chances of developing pancytopenia?

Pancytopenia is not extremely uncommon following SCT with a median time of occurrence of 20 months. Most of the cases occurred in the early period of SCT and resolved themselves. However, some might have taken longer to resolve. Thus, if any clinical problem ensues, it might be wise to get a bone marrow examination.

Anonymous Patient Answer
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