| Factors Influencing Outcome and Incidence of Late Complications in Children who Underwent Allogeneic Hematopoietic Stem Cell Transplantation for Hemoglobinopathy. | |
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MedLine Citation:
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PMID: 23020512 Owner: NLM Status: Publisher |
Abstract/OtherAbstract:
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Background: Hematopoietic stem cell transplantation (HSCT) remains the only potentially curative treatment for severe hemoglobinopathy (HGP). Late complications (LCs) are all events occurring beyond two years post-HSCT. We retrospectively analyzed prevalence, factors influencing occurrence, and prognosis of LCs post-HSCT for HGP. Patients and Methods: Between 2000 and 2011, 47 patients (21 males, 26 females; 43 with beta thalassemia major, four with sickle cell disease) who had survived more than two years post-HSCT for HGP were retrospectively reviewed. Mean age at HSCT was 7.7 years (1.1-32 years); mean follow-up was 7.1 years (2-11.6 years); 11 patients were splenectomized; mean ferritin level was 3022 ng/mL (350-10900); and seven patients underwent a second HSCT. Results: Endocrinological complications were observed with primary gonadal failure in 16/20 mature females and 4/11 mature males, in five patients with primary hypothyroidism and in four with insulin-dependent diabetes mellitus (DM). Skeletal complications were observed in 10 with secondary osteoporosis; 22 patients had elevated transaminase levels; two had hepatitis B reactivation. Neurological, cardiac and ocular manifestations were relatively rare. A higher incidence of LCs was observed in splenectomized than in nonsplenectomized patients: cGVHD -64% versus 13% (P = .003); endocrine abnormalities -91% versus 30.5%, (P = .001); elevated transaminase levels -73% versus 33% (P = .043); mortality -18% versus 2.7% (NS). Conclusions: LCs post-HSCT for HGP are common and heterogeneous. Etiology is multifactorial with iron overload (IO), class, splenectomy, age, chronic GVHD, and corticosteroid (CS) treatment. Our data may help build follow-up guidelines to limit, detect, and treat any LCs and improve quality of life. |
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Authors:
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Abdalla Khalil; Irena Zaidman; Ronit Elhasid; Monique Peretz-Nahum; Boris Futerman; Myriam Ben-Arush |
Publication Detail:
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Type: JOURNAL ARTICLE Date: 2012-9-28 |
Journal Detail:
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Title: Pediatric hematology and oncology Volume: - ISSN: 1521-0669 ISO Abbreviation: Pediatr Hematol Oncol Publication Date: 2012 Sep |
Date Detail:
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Created Date: 2012-10-1 Completed Date: - Revised Date: - |
Medline Journal Info:
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Nlm Unique ID: 8700164 Medline TA: Pediatr Hematol Oncol Country: - |
Other Details:
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Languages: ENG Pagination: - Citation Subset: - |
Affiliation:
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1Department of Pediatric Hematology Oncology, Meyer Children's Hospital, Rambam Health Care Campus, Haifa, Israel. |
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From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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