TY - JOUR
T1 - Factors influencing outcome and incidence of late complications in children who underwent allogeneic hematopoietic stem cell transplantation for hemoglobinopathy
AU - Khalil, Abdalla
AU - Zaidman, Irena
AU - Elhasid, Ronit
AU - Peretz-Nahum, Monique
AU - Futerman, Boris
AU - Ben-Arush, Myriam
PY - 2012/11
Y1 - 2012/11
N2 - 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 (1.132 years); mean follow-up was 7.1 years (211.6 years); 11 patients were splenectomized; mean ferritin level was 3022 ng/mL (35010900); 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.
AB - 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 (1.132 years); mean follow-up was 7.1 years (211.6 years); 11 patients were splenectomized; mean ferritin level was 3022 ng/mL (35010900); 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.
KW - Allogeneic hematopoietic stem cell transplantation
KW - Hemoglobinopathy
KW - Late complications
KW - Sickle cell disease
UR - http://www.scopus.com/inward/record.url?scp=84867952034&partnerID=8YFLogxK
U2 - 10.3109/08880018.2012.725198
DO - 10.3109/08880018.2012.725198
M3 - ???researchoutput.researchoutputtypes.contributiontojournal.article???
C2 - 23020512
AN - SCOPUS:84867952034
SN - 0888-0018
VL - 29
SP - 694
EP - 703
JO - Pediatric Hematology and Oncology
JF - Pediatric Hematology and Oncology
IS - 8
ER -