Evidence-Based Minireview: In young children with severe sickle cell disease, do the benefits of HLA-identical sibling donor HCT outweigh the risks?

Abstract

In case 1, a 14 - month - old male child with sickle cell dis ease (SCD) was referred for eval u a tion for an allo ge neic hema to poietic stem cell trans plant (HCT). The patient had a his tory of dactylitis 3 times in his fi rst year of life and febrile epi sodes twice at the con sult. His 4 - year - old sis ter was found to be human leu ko cyte anti gen (HLA) iden ti cal. The patient was started on hydroxy urea (HU) at 2.5 years of age. His parents again sought con sul ta tion when he was 5 years old because of con cerns about his med i cal con di tion. At the time, the patient had expe ri enced 2 vaso - occlu sive pain epi sodes (VOEs) requir ing hos pi tal i za tion dur ing the pre vi ous 2 years. He had also expe ri enced inter mit tent pain cri ses requir ing rest at home for 2 to 3 days. The child has not attended school in per son due to the COVID - 19 pan demic. The fam ily is con sid er ing HCT but is ambiv a lent about it because of poten tial tox ic ity. In case 2, an 8 - year - old female child is 3 years out from HCT for SCD from her HLA - iden ti cal sib ling. Before HCT, despite receiv ing HU, she had expe ri enced > 5 VOEs requir ing hos pi tali za tion and 2 epi sodes of acute chest syn dromes in the pre vi ous 3 years. She had also been miss ing almost 50 days of school days each year. After HCT, she is now attend ing school reg u larly and par tici pat ing in all nor mal age - appro pri ate activ i ties. The par ents believe that HCT has been trans for ma tive in their child ' s life.