Document Detail

Factors Influencing Outcome and Incidence of Late Complications in Children who Underwent Allogeneic Hematopoietic Stem Cell Transplantation for Hemoglobinopathy.
MedLine Citation:
PMID:  23020512     Owner:  NLM     Status:  Publisher    
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.
Abdalla Khalil; Irena Zaidman; Ronit Elhasid; Monique Peretz-Nahum; Boris Futerman; Myriam Ben-Arush
Publication Detail:
Type:  JOURNAL ARTICLE     Date:  2012-9-28
Journal Detail:
Title:  Pediatric hematology and oncology     Volume:  -     ISSN:  1521-0669     ISO Abbreviation:  Pediatr Hematol Oncol     Publication Date:  2012 Sep 
Date Detail:
Created Date:  2012-10-1     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  8700164     Medline TA:  Pediatr Hematol Oncol     Country:  -    
Other Details:
Languages:  ENG     Pagination:  -     Citation Subset:  -    
1Department of Pediatric Hematology Oncology, Meyer Children's Hospital, Rambam Health Care Campus, Haifa, Israel.
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