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About 1,830 results

ALLMedicine™ Fanconi Anemia Center

Research & Reviews  689 results

Eltrombopag for People With Fanconi Anemia
https://clinicaltrials.gov/ct2/show/NCT03206086

May 13th, 2022 - Fanconi anemia (FA) is a rare genetic disease that often presents as a bone marrow failure (BMF) syndrome but also can affect any other organ. Etiologically, loss of function mutations in more than 21 different gene members of the FA core complex ...

Cancer in Inherited Bone Marrow Failure Syndromes
https://clinicaltrials.gov/ct2/show/NCT00027274

May 13th, 2022 - Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders. Pathogenic variant(s) in IBMFS genes are relevant to carcinogenesis in sporadic ca...

Metformin for Treatment of Cytopenias in Children and Young Adults with Fanconi Anemia.
https://doi.org/10.1182/bloodadvances.2021006490
Blood Advances; Pollard J, Furutani E et. al.

May 3rd, 2022 - Fanconi anemia (FA), a genetic disorder affecting DNA repair, is characterized by bone marrow failure and cancer susceptibility. In FA mouse models the biguanide metabolic agent metformin improves blood counts and delays tumor development. We cond...

Cell senescence and malignant transformation in the inherited bone marrow failure syndr...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9062194
Seminars in Hematology; Groarke EM, Calado RT et. al.

May 2nd, 2022 - Fanconi anemia, telomeropathies and ribosomopathies are members of the inherited bone marrow failure syndromes, rare genetic disorders that lead to failure of hematopoiesis, developmental abnormalities, and cancer predisposition. While each disord...

Distinct clinical and somatic mutational features of breast tumors with high-, low-, or...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9052533
BMC Medicine; Zhang G, Ren C et. al.

Apr 29th, 2022 - HER2-low breast cancers were reported to have distinct clinicopathological characteristics from HER2-zero; however, the difference in their genetic features remains unclear. This study investigated the clinical and molecular features of breast tum...

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Clinicaltrials.gov  23 results

Eltrombopag for People With Fanconi Anemia
https://clinicaltrials.gov/ct2/show/NCT03206086

May 13th, 2022 - Fanconi anemia (FA) is a rare genetic disease that often presents as a bone marrow failure (BMF) syndrome but also can affect any other organ. Etiologically, loss of function mutations in more than 21 different gene members of the FA core complex ...

Cancer in Inherited Bone Marrow Failure Syndromes
https://clinicaltrials.gov/ct2/show/NCT00027274

May 13th, 2022 - Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders. Pathogenic variant(s) in IBMFS genes are relevant to carcinogenesis in sporadic ca...

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
https://clinicaltrials.gov/ct2/show/NCT02143830

Apr 8th, 2022 - The trial proposed is a three arm phase II treatment protocol designed to investigate the safety and efficacy of risk-adjusted chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem ce...

Gene Therapy for Fanconi Anemia
https://clinicaltrials.gov/ct2/show/NCT01331018

Dec 15th, 2021 - PRIMARY OBJECTIVE: I. To determine the safety of lentiviral gene transfer for patients with Fanconi anemia complementation group A. SECONDARY OBJECTIVES: I. To determine the feasibility and efficacy of filgrastim (G-CSF) and plerixafor mobilizatio...

Sirolimus, Docetaxel, and Carboplatin in Treating Patients With Metastatic Castration-Resistant Prostate Cancer
https://clinicaltrials.gov/ct2/show/NCT02565901

Oct 1st, 2021 - PRIMARY OBJECTIVES: I. To define the recommended phase 2 dose (RP2D) of sirolimus combined with docetaxel and carboplatin in the treatment of metastatic castration resistant prostate cancer (CRPC). (Phase I) II. To assess the effectiveness of siro...

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News  18 results

PARP Inhibition Arrives in Pancreatic Cancer
https://www.onclive.com/view/parp-inhibition-arrives-in-pancreatic-cancer

Oct 6th, 2021 - Michael J. Hall, MD PARP inhibitors, which have approved indications in breast and ovarian cancer, are expanding to additional treatment paradigms, including pancreatic cancer. Data from the phase III POLO trial presented at the 2019 ASCO Annual...

Improvements in Detecting and Treating Metastatic CRPC
https://www.onclive.com/view/improvements-in-detecting-and-treating-metastatic-crpc

Apr 2nd, 2021 - Transcript: Raoul S. Concepcion, MD, FACS: I think we’ve all talked about this. We’ve seen this discussed in multiple publications. There have been multiple discussion points about these patients who progress to true metastatic castration-resi...

PARP Inhibitors in BRCA1/2-mutated Patients in Prostate Cancer
https://www.onclive.com/view/parp-inhibitors-in-brca1-2-mutated-patients-in-prostate-cancer

Apr 2nd, 2021 - Transcript:Raoul S. Concepcion, MD: Moving on to our next segment, one of the interesting articles that has been published this year is the Pritchard article that was in The New England Journal of Medicine, which basically looked at DNA repair gen...

Mechanisms of Immunotherapy in HNSCC
https://www.onclive.com/view/mechanisms-of-immunotherapy-in-hnscc

Apr 2nd, 2021 - Transcript:Robert L. Ferris, MD, PhD: Cetuximab is a monoclonal antibody. It was developed to block the epidermal growth factor receptor, or EGFR. The higher the level of EGFR, the worse the clinical outcome. Therefore, it made sense to use a bloc...

FDA Approval in Myeloma, Label Update in HER2+ Breast Cancer, Breakthrough Status in Prostate Cancer, and More
https://www.onclive.com/view/fda-approval-in-myeloma-label-update-in-her2-breast-cancer-breakthrough-status-in-prostate-cancer-and-more

Apr 2nd, 2021 - Today- An FDA approval in multiple myeloma, a label update in breast cancer, a breakthrough therapy designation in prostate cancer, research grants awarded in rare diseases, and two trials in pediatric acute lymphoblastic leukemia stopped early...

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Patient Education  2 results see all →