Factors influencing outcome and incidence of late complications in children who underwent allogeneic hematopoietic stem cell transplantation for hemoglobinopathy

Abdalla Khalil, Irena Zaidman, Ronit Elhasid, Monique Peretz-Nahum, Boris Futerman, Myriam Ben-Arush*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

17 Scopus citations

Abstract

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.

Original languageEnglish
Pages (from-to)694-703
Number of pages10
JournalPediatric Hematology and Oncology
Volume29
Issue number8
DOIs
StatePublished - Nov 2012
Externally publishedYes

Keywords

  • Allogeneic hematopoietic stem cell transplantation
  • Hemoglobinopathy
  • Late complications
  • Sickle cell disease

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