ATRA Plus G-CSF for Mobilization of Hematopoietic Stem and Progenitor Cells

2014-08-27 03:41:33 | BioPortfolio


Hematopoietic stem and progenitor cells (HSPC) are used for transplantation in patients undergoing high dose therapy for the treatment of a range of cancers.

- HSPC are collected from the bloodstream after treatment with medications that cause the HSPC to move from the bone marrow into the bloodstream, a process called mobilization

- between 5 and 60% of patients can fail to collect enough HSPC for a transplant, using current mobilization techniques

- this study aims to assess the safety of combining a derivative of vitamin A, ATRA with G-CSF (the drug most commonly used to mobilize HSPC)

- ATRA has never been combined with G-CSF for mobilization of HSPC and therefore a study is needed to assess the safety of this combination, and whether it successfully mobilizes HSPC


HSPC mobilization is normally achieved using cytokines such as G-CSF, or occasionally GM-CSF, often in combination with myelosuppressive chemotherapy.

Studies in the mouse model have shown that retinoids (vitamin A derivatives) can be combined with G-CSF, and that this combination synergizes to enhance HSPC mobilization over that seen with G-CSF alone.

This trial aims to assess the safety and mobilization efficacy of combining mobilizing doses of G-CSF with a standard dose of ATRA, using a treatment regimen derived from the earlier murine studies.

In this phase I pilot study, six patients with multiple myeloma or cutaneous lymphoma will be treated with ATRA plus G-CSF, and safety and toxicity data collected for the two week study drug period plus a further two weeks' follow-up. The primary endpoint is safety and toxicity, the secondary endpoint is an observation of the mobilization efficacy as demonstrated by CD34+ cell counts over the study period. Patients will not undergo stem cell collection during this study, as this is purely an observational study. Participating patients will not be those who would normally be scheduled for stem cell collection and transplantation in the near future, but rather patients with stable disease who are not candidates for imminent transplantation, or who have collected adequate HSPC on previous mobilization attempts and are currently being observed.

Cutaneous lymphoma and multiple myeloma are chosen as suitable disease states for this study as there is in vitro evidence of a possible disease benefit of retinoids in these disorders. If disease response is noted during the study or follow up period, ongoing ATRA will be offered at the discretion of the treating physician.

Study Design

Allocation: Non-Randomized, Control: Uncontrolled, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Multiple Myeloma


ATRA plus G-CSF (filgrastim, NEUPOGEN (R)) combination


Peter MacCallum Cancer Center
East Melbourne




Peter MacCallum Cancer Centre, Australia

Results (where available)

View Results


Published on BioPortfolio: 2014-08-27T03:41:33-0400

Clinical Trials [1820 Associated Clinical Trials listed on BioPortfolio]

ATRA, Celecoxib, and Itraconazole as Maintenance

The purpose of this study is to evaluate the safety and tolerability associated with the combination of ATRA/celecoxib/itraconazole as maintenance therapy given after an autologous stem ce...

A Research Study To Test How Multiple Doses Of Filgrastim Hospira Works In The Body Of Healthy Study Subjects When Given by Subcutaneous Injection (SC) (Shot) Compared To An Already U.S.-Approved Drug Neupogen® (Amgen)

This is a study comparing two study drugs, Filgrastim Hospira and Neupogen®. Neupogen® is approved by the US Food and Drug Administration (FDA) to treat low numbers of specific kinds of ...

Post-ASCT Use of Zarzio®

There are limited data concerning the use of biosimilars of filgrastim in autologous stem cell transplantation (ASCT). This study aimed to evaluate G-CSF efficiency and safety (based on ha...

S9922 Combination Chemo Plus Filgrastim With or Without Thalidomide in Refractory Multiple Myeloma

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Thalidomide may stop the growth of tumor cells by stopping blood fl...

Melphalan and Filgrastim to Stimulate Peripheral Stem Cells in Patients With Multiple Myeloma

RATIONALE: Melphalan and colony-stimulating factors such as filgrastim may increase the number of immune cells found in bone marrow or peripheral blood. PURPOSE: Phase II trial to study t...

PubMed Articles [6160 Associated PubMed Articles listed on BioPortfolio]

Severe Cytomegalovirus Enterocolitis Developing following Daratumumab Exposure in Three Patients with Multiple Myeloma.

Survival of multiple myeloma (MM) patients has improved with introduction of novel anti-myeloma agents. Myeloma has transformed into a chronic condition, accompanied with multiple relapses requiring s...

Identification specific miRNA in t(4;14) multiple myeloma based on miRNA-mRNA expressing profile correlation analysis.

Multiple myeloma (MM) is a common malignancy belonging to the hematological system. The translocation t(4;14)(p16.3;q32.3) is a critical cytogenetic change of MM, which is presenting a poor prognosis....

Serum protein N-glycosylation changes in multiple myeloma.

Multiple myeloma is characterized by clonal proliferation of malignant plasma cells in the bone marrow that produce monoclonal immunoglobulins. N-glycosylation changes of these monoclonal immunoglobul...

A clinical prediction model for outcome and therapy delivery in transplant-ineligible patients with myeloma (UK Myeloma Research Alliance Risk Profile): a development and validation study.

Tolerability of treatments for multiple myeloma can depend on the characteristics of the patient being treated. We aimed to develop and validate a risk profile, using routinely collected data, that co...

PRC2 targeting is a therapeutic strategy for EZ score defined high-risk multiple myeloma patients and overcome resistance to IMiDs.

Multiple myeloma (MM) is a malignant plasma cell disease with a poor survival, characterized by the accumulation of myeloma cells (MMCs) within the bone marrow. Epigenetic modifications in MM are asso...

Medical and Biotech [MESH] Definitions

An asymptomatic and slow-growing PLASMA CELL dyscrasia characterized by presence of MYELOMA PROTEINS and clonal bone marrow plasma cells without end-organ damage (e.g., renal impairment). It is distinguished from MONOCLONAL GAMMOPATHY OF UNDETERMINED SIGNIFICANCE by a much higher risk of progression to symptomatic MULTIPLE MYELOMA.

A rare, aggressive variant of MULTIPLE MYELOMA characterized by the circulation of excessive PLASMA CELLS in the peripheral blood. It can be a primary manifestation of multiple myeloma or develop as a terminal complication during the disease.

Abnormal immunoglobulins characteristic of MULTIPLE MYELOMA.

An abnormal protein with unusual thermosolubility characteristics that is found in the urine of patients with MULTIPLE MYELOMA.

A pyrazine and boronic acid derivative that functions as a reversible PROTEASOME INHIBITOR. It is used as an ANTINEOPLASTIC AGENT in the treatment of MULTIPLE MYELOMA and MANTLE CELL LYMPHOMA.

More From BioPortfolio on "ATRA Plus G-CSF for Mobilization of Hematopoietic Stem and Progenitor Cells"

Quick Search


Relevant Topics

Organ transplantation is the moving of an organ from one body to another or from a donor site to another location on the patient's own body, for the purpose of replacing the recipient's damaged or absent organ. The emerging field of regenerative ...

Osteoporosis is a disease in which the bones become extremely porous, are subject to fracture, and heal slowly, occurring especially in women following menopause and often leading to curvature of the spine from vertebral collapse. Follow and track&n...

Within medicine, nutrition (the study of food and the effect of its components on the body) has many different roles. Appropriate nutrition can help prevent certain diseases, or treat others. In critically ill patients, artificial feeding by tubes need t...

Searches Linking to this Trial