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About 1,843 results

ALLMedicine™ Fanconi Anemia Center

Research & Reviews  690 results

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...

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 ...

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  26 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...

Trial of Maintenance With Niraparib- Uterine Serous Carcinoma
https://clinicaltrials.gov/ct2/show/NCT04080284

Mar 18th, 2022 - Uterine serous carcinoma (USC) accounts for up to 40% of endometrial cancer-related deaths. In contrast to the more common endometrioid histology, USC is more likely to present in advanced stage and carries a worse prognosis. USC mimics the most c...

Umbilical Cord Blood Infusion to Treat Type 1 Diabetes
https://clinicaltrials.gov/ct2/show/NCT00305344

Feb 9th, 2022 - Objective: Our goal is to transfuse autologous umbilical cord blood into 23 children with T1D in an attempt to re-establish immune tolerance and perhaps regenerate pancreatic islet insulin-producing beta cells and improve blood glucose control. As...

Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor
https://clinicaltrials.gov/ct2/show/NCT02678533

Dec 22nd, 2021 - Fanconi anemia is an autosomal recessive disease with an average survival of around 24 years old. The number of cells producted by bone marrow decreases around 5-10 years old. Hematological symptoms occur around 7 years old. 80% of patients with F...

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News  19 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 →