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Survival and Late E...
Survival and Late Effects after Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancy at Less than Three Years of Age
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- Vrooman, Lynda M. (author)
- Dana Farber Boston Childrens Canc & Blood Disorde, Dept Pediat, 450 Brookline Ave, Boston, MA 02215 USA
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- Millard, Heather R. (author)
- Med Coll Wisconsin, Dept Med, Ctr Int Blood & Marrow Transplant Res, Milwaukee, WI 53226 USA
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- Brazauskas, Ruta (author)
- Med Coll Wisconsin, Dept Med, Ctr Int Blood & Marrow Transplant Res, Milwaukee, WI 53226 USA;Med Coll Wisconsin, Div Biostat, Inst Hlth & Soc, Milwaukee, WI 53226 USA
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- Majhail, Navneet S. (author)
- Cleveland Clin, Taussig Canc Inst, Blood & Marrow Transplant Program, Cleveland, OH 44106 USA
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- Battiwalla, Minoo (author)
- NHLBI, Hematol Branch, Bethesda, MD 20892 USA
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- Flowers, Mary E. (author)
- Fred Hutchinson Canc Res Ctr, Div Clin Res, Seattle, WA 98104 USA
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- Savani, Bipin N. (author)
- Vanderbilt Univ, Med Ctr, Div Hematol Oncol, Dept Med, Nashville, TN 37235 USA
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- Akpek, Gorgun (author)
- Rush Univ, Med Ctr, Dept Internal Med, Stem Cell Transplantat & Cell Therapy, Chicago, IL 60612 USA
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- Aljurf, Mahmoud (author)
- King Faisal Specialist Hosp Ctr & Res, Dept Oncol, Riyadh, Saudi Arabia
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- Bajwa, Rajinder (author)
- Nationwide Childrens Hosp, Div Hematol Oncol BMT, Columbus, OH USA
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- Baker, K. Scott (author)
- Fred Hutchinson Canc Res Ctr, Div Clin Res, Seattle, WA 98104 USA
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- Beitinjaneh, Amer (author)
- Univ Miami, Div Hematol & Oncol, Miami, FL USA
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- Bitan, Menachem (author)
- Tel Aviv Sourasky Med Ctr, Dept Pediat Hematol Oncol, Tel Aviv, Israel
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- Buchbinder, David (author)
- Childrens Hosp Orange Cty, Div Hematol, Orange, CA 92668 USA
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- Chow, Eric (author)
- Fred Hutchinson Canc Res Ctr, Div Clin Res, Seattle, WA 98104 USA
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- Dandoy, Christopher (author)
- Cincinnati Childrens Hosp Med Ctr, Div Bone Marrow Transplant & Immune Deficiency, Cincinnati, OH 45229 USA
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- Dietz, Andrew C. (author)
- Univ Southern Calif, Childrens Hosp Los Angeles, Div Hematol Oncol, Blood & Marrow Transplantat, Los Angeles, CA USA
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- Diller, Lisa (author)
- Dana Farber Boston Childrens Canc & Blood Disorde, Dept Pediat, 450 Brookline Ave, Boston, MA 02215 USA
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- Gale, Robert Peter (author)
- Imperial Coll London, Hematol Res Ctr, Dept Med, Div Expt Med, London, England
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- Hashmi, Shahrukh K. (author)
- Mayo Clin, Div Hematol, Dept Internal Med, Rochester, MN USA
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- Hayashi, Roberti (author)
- Washington Univ, Sch Med, Div Pediat Hematol Oncol, Dept Pediat, St Louis, MO USA
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- Hematti, Peiman (author)
- Univ Wisconsin Hosp & Clin, Div Hematol Oncol Bone Marrow Transplantat, Dept Med, Madison, WI 53792 USA
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- Kamble, Rammurti T. f (author)
- Baylor Coll Med, Div Hematol & Oncol, Ctr Cell & Gene Therapy, Houston, TX 77030 USA
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- Kasow, Kimberly A. (author)
- Univ North Carolina Chapel Hill, Div Hematol Oncol, Dept Pediat, Chapel Hill, NC USA
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- Kletzel, Morris (author)
- Ann & Robert H Lurie Childrens Hosp Chicago, Div Hematol Oncol & Stem Cell Transplant, Dept Pediat, Chicago, IL 60611 USA
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- Lazarus, Hillard M. (author)
- Univ Hosp Cleveland, Med Ctr, Div Hematol & Oncol, Seidman Canc Ctr, Cleveland, OH 44106 USA
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- Malone, Adriana K. (author)
- Icahn Sch Med Mt Sinai, Div Hematol Oncol, Tisch Canc Inst, New York, NY 10029 USA
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- Marks, David I. (author)
- Univ Hosp Bristol NHS Trust, Adult Bone Marrow Transplant, Bristol, Avon, England
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- O'Brien, Tracey A. (author)
- Sydney Childrens Hosp, Kids Canc Ctr, Blood & Marrow Transplant Program, Sydney, NSW, Australia
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- Olsson, Richard (author)
- Karolinska Institutet,Uppsala universitet,Centrum för klinisk forskning i Sörmland (CKFD),Karolinska Inst, Div Therapeut Immunol, Dept Lab Med, Stockholm, Sweden
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- Ringden, Olle (author)
- Karolinska Institutet
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- Seo, Sachiko (author)
- East Hosp, Natl Canc Res Ctr, Kashiwa, Chiba, Japan
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- Steinberg, Amir (author)
- Icahn Sch Med Mt Sinai, Div Hematol Oncol, Tisch Canc Inst, New York, NY 10029 USA
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- Yu, Lolie C. (author)
- Louisiana State Univ, Childrens Hosp, Med Ctr, Div Hematol Oncol,Ctr Canc & Blood Disorders, New Orleans, LA USA
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- Warwick, Anne (author)
- Uniformed Serv Univ Hlth Sci, Dept Pediat, Bethesda, MD 20814 USA
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- Shaw, Bronwen (author)
- Med Coll Wisconsin, Dept Med, Ctr Int Blood & Marrow Transplant Res, Milwaukee, WI 53226 USA
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- Duncan, Christine (author)
- Dana Farber Boston Childrens Canc & Blood Disorde, Dept Pediat, 450 Brookline Ave, Boston, MA 02215 USA
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(creator_code:org_t)
- Elsevier BV, 2017
- 2017
- English.
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In: Biology of blood and marrow transplantation. - : Elsevier BV. - 1083-8791 .- 1523-6536. ; 23:8, s. 1327-1334
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Abstract
Subject headings
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- Very young children undergoing hematopoietic cell transplantation (HCT) are a unique and vulnerable population. We analyzed outcomes of 717 patients from 117 centers who survived relapse free for year after allogeneic myeloablative HCT for hematologic malignancy at <3 years of age, between 1987 and 2012. The median follow-up was 8.3 years (range, 1.0 to 26.4 years); median age at follow-up was 9 years (range, 2 to 29 years). Ten-year overall and relapse-free survival were 87% (95% confidence interval [CI], 85% to 90%) and 84% (95% CI, 81% to 87%). Ten-year cumulative incidence of relapse was 11% (95% CI, 9% to 13%). Of 84 deaths, relapse was the leading cause (43%). Chronic graft-versus-host-disease 1 year after HCT was associated with increased risk of mortality (hazard ratio [HR], 2.1; 95% CI, 1.3 to 3.3; P=.0018). Thirty percent of patients experienced >= 1 organ toxicity/late effect >1 year after HCT. The most frequent late effects included growth hormone deficiency/growth disturbance (10-year cumulative incidence, 23%; 95% CI, 19% to 28%), cataracts (18%; 95% CI, 15% to 22%), hypothyroidism (13%; 95% CI, 10% to 16%), gonadal dysfunction/infertility requiring hormone replacement (3%; 95% CI, 2% to 5%), and stroke/seizure (3%; 95% CI, 2% to 5%). Subsequent malignancy was reported in 3.6%. In multivariable analysis, total body irradiation (TBI) was predictive of increased risk of cataracts (HR, 17.2; 95% CI, 7.4 to 39.8; P <.001), growth deficiency (HR, 3.5; 95% CI, 2.2 to 5.5; P <.001), and hypothyroidism (HR, 5.3; 95% CI, 3.0 to 9.4; P <.001). In summary, those who survived relapse free year after HCT for hematologic malignancy at <3 years of age had favorable overall survival. Chronic graft-versus host-disease and TBI were associated with adverse outcomes. Future efforts should focus on reducing the risk of relapse and late effects after HCT at early age.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Hematologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Hematology (hsv//eng)
Keyword
- Hematopoietic cell transplantation (HCT)
- Survival
- Infants
- Pediatric
- Late effects
- Hematologic malignancy
- Relapse
- Total body irradiation
- Graft-versus-host disease
Publication and Content Type
- ref (subject category)
- art (subject category)
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- By the author/editor
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Vrooman, Lynda M ...
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Millard, Heather ...
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Brazauskas, Ruta
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Majhail, Navneet ...
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Battiwalla, Mino ...
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Flowers, Mary E.
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show more...
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Savani, Bipin N.
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Akpek, Gorgun
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Aljurf, Mahmoud
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Bajwa, Rajinder
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Baker, K. Scott
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Beitinjaneh, Ame ...
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Bitan, Menachem
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Buchbinder, Davi ...
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Chow, Eric
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Dandoy, Christop ...
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Dietz, Andrew C.
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Diller, Lisa
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Gale, Robert Pet ...
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Hashmi, Shahrukh ...
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Hayashi, Roberti
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Hematti, Peiman
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Kamble, Rammurti ...
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Kasow, Kimberly ...
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Kletzel, Morris
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Lazarus, Hillard ...
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Malone, Adriana ...
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Marks, David I.
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O'Brien, Tracey ...
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Olsson, Richard
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Ringden, Olle
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Seo, Sachiko
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Steinberg, Amir
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Yu, Lolie C.
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Warwick, Anne
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Shaw, Bronwen
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Duncan, Christin ...
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- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Hematology
- Articles in the publication
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Biology of blood ...
- By the university
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Uppsala University
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Karolinska Institutet