Hematopoiesis and Bone Marrow Failure

Research in the Hematopoiesis and Bone Marrow Failure Laboratory, led by Dr. Neal Young, spans the basic sciences, clinical trials, and epidemiology. Bench work involves methods of cell and molecular biology, immunology, and virology.

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Neal Young

Clinical Trials and Studies

Danazol en dosis bajas para el tratamiento de enfermedades relacionadas al telómero

RECLUTAMIENTO
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Algunas personas que tienen anemia aplásica tienen telómeros muy cortos, que protege los extremos del ADN en los cromosomas. Este estudio analiza si dosis bajas del medicamento danazol ayudan a prevenir el acortamiento de los telómeros y a reducir los signos de daño por la anemia aplásica o afecciones relacionadas. Los participantes de este estudio deben tener 3 años o más, deben tener enfermedad de los telómeros y signos de anemia aplásica, enfermedad pulmonar como fibrosis pulmonar o enfermedad hepática. Este estudio se lleva a cabo en Bethesda (Maryland).

Investigation of the Natural Progression of Clonal Hematopoiesis of Indeterminate Potential and Clonal Cytopenia of Undetermined Significance.

Recruiting
Adult, Older Adult
All Genders
Not Accepting Healthy Volunteers
Are you an adult with clonal hematopoiesis of indeterminate potential (CHIP) or clonal cytopenia of undetermined significance (CCUS)? This study is working with people who have CHIP and CCUS to determine whether they have changes in their genes that raise the risk of atherosclerosis or blood cancers. To participate in this study, you must be at least 18 years old with CHIP or CCUS. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

Recruiting
All Ages
All Genders
Not Accepting Healthy Volunteers
Do you or your child have bone marrow failure disease or a telomere biology disorder? This study looks at the long-term effects of bone marrow failure disease and its treatments on organ function. Participants in this study must be 2 years or older with bone marrow failure or telomere biology disorder. This study is located at the NIH Clinical Center in Bethesda, Maryland.

JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure

Recruiting
Adult, Older Adult
All Genders
Not Accepting Healthy Volunteers
Are you aged 18 and older with an immune bone marrow failure? This study is testing a drug called ruxolitinib in people with different types of immune bone marrow failure. Participants will have blood tests every week while they are taking the drug. These tests can be done by the participant s own physician and the results sent to the researchers. This study will take place at the National Institutes of Health Clinical Center in Bethesda, Maryland.

Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)

Recruiting
All Ages
All Genders
Not Accepting Healthy Volunteers
Do you or your child have severe aplastic anemia? This study tests the safety and effectiveness of two medicines called cyclosporine and eltrombopag in people with severe aplastic anemia. The typical treatment is a combination of three medicines: cyclosporine, eltrombopag, and horse anti-thymocyte globulin. Researchers want to see if starting people at a lower dose of cyclosporine and eltrombopag before giving them horse anti-thymocyte globulin is helpful. Participants in this study must be 4 years or older with severe aplastic anemia. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Collection of Blood, Bone Marrow, and Buccal Mucosa Samples from Healthy Volunteers

Recruiting
All Ages
All Genders
Accepting Healthy Volunteers
Are you or your child willing to donate blood or bone marrow to help research? This study collects samples of blood, bone marrow, and buccal mucosa (cells from the inside of the cheek). Researchers will use these samples to study blood cells, blood diseases, and the role of the immune system in conditions such as aplastic anemia. Participants in this study must be at least 8 years old. This study is located at the NIH Clinical Center in Bethesda, Maryland.

Eltrombopag con inmunosupresión estándar para la anemia aplásica grave

RECLUTAMIENTO
Todas las edades
All
No Se Aceptan Voluntaries Saludables
Este estudio analiza si el agregado del medicamento eltrombopag a los tratamientos estándar es una mejor manera de tratar la anemia aplásica grave. Los participantes de este estudio deben tener 2 años o más, además de anemia aplásica grave que todavía no ha recibido tratamiento. Este estudio se lleva a cabo en Bethesda (Maryland).

Featured Publications

Eltrombopag added to immunosuppression for children with treatment-naïve severe aplastic anaemia.

Authors: Groarke EM, Patel BA, Gutierrez-Rodrigues F, Rios O, Lotter J, Baldoni D, St Pierre A, Shalhoub R, Wu CO, Townsley DM, Young NS.
Br J Haematol. 2021 Feb 01;192(3):605-514
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HLA associations, somatic loss of HLA expression, and clinical outcomes in immune aplastic anemia.

Authors: Zaimoku Y, Patel BA, Adams SD, Shalhoub R, Groarke EM, Lee AAC, Kajigaya S, Feng X, Rios OJ, Eager H, Alemu L, Quinones Raffo D, Wu CO, Flegel WA, Young NS.
Blood. 2021 Dec 30;138(26):2799-2809
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Long-term outcomes in patients with severe aplastic anemia treated with immunosuppression and eltrombopag: a phase 2 study.

Authors: Patel BA, Groarke EM, Lotter J, Shalhoub R, Gutierrez-Rodrigues F, Rios O, Quinones Raffo D, Wu CO, Young NS.
Blood. 2022 Jan 06;139(1):34-43
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Benign and malignant hematologic manifestations in patients with VEXAS syndrome due to somatic mutations in UBA1.

Authors: Obiorah IE, Patel BA, Groarke EM, Wang W, Trick M, Ombrello AK, Ferrada MA, Wu Z, Gutierrez-Rodrigues F, Lotter J, Wilson L, Hoffmann P, Cardona DO, Patel N, Dulau-Florea A, Kastner DL, Grayson PC, Beck DB, Young NS, Calvo KR.
Blood Adv. 2021 Aug 24;5(16):3203-3215
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Somatic Mutations in "Benign" Disease.

Authors: Mustjoki S, Young NS.
N Engl J Med. 2021 May 27;384(21):2039-2052
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Meet the Team

Neal Young

Neal Young, M.D.

Senior Investigator

Neal Young received a A.B. cum laude from Harvard College in 1967 and his M.D. in 1971 from the Johns Hopkins School of Medicine. He did post-graduate medical training at Massachusetts General Hospital and Barnes Hospital at Washington University in St. Louis before joining the NHLBI in 1981. He is a Master of the American College of Physicians and has accrued many academic honors. His efforts as a US Government employee were recognized with Samuel J. Heyman Service to America Medal in Science and the Environment. Dr. Young has authored over 280 original scientific and medical articles in peer reviewed journals and more than 160 reviews and book chapters. He also is author or editor of 10 medical and scientific books, including a new textbook of hematology. Dr. Young is a member of the American Society for Clinical Investigation, American Association of Physicians, American Federation for Clinical Research, American Society of Hematology, and International Society for Experimental Hematology. He is co-inventor on 7 patents concerning B19 parvovirus. Dr. Young’s trainees head departments of hematology worldwide. He has lectured and published prolifically in hematology. He led a novel training program in Vietnam that transformed that country’s approach to blood diseases. He now heads a successful new trans-NIH initiative in human immunology.

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Kate Roskom

Research Nurse
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