A case report on the treatment of autoimmune hemolytic anemia after CBT by daratumumab in a mucopolysaccharidosis type III patient.

rationaleMucopolysaccharidosis type III, also referred to as Sanfilippo disease, is a rare hereditary lysosomal storage illness and one of the more common variants. The illness can affect all tissues and organs of the body, with the central nervous system being the most profoundly impacted. Autoimmune hemolytic anemia (AIHA) is a rare, diverse, antibody-mediated disorder characterized by the production of antibodies that target and destroy red blood cells, leading to their rapid destruction. AIHA is a prevalent and notable complication in recipients following umbilical cord blood stem cell transplantation. Immunosuppressive treatments, including intravenous immunoglobulin and rituximab, have been the primary therapeutic modalities. There is no consensus on the appropriate treatment for post-transplant severe AIHA. PATIENT CONCERNS: This article details a 5-year-old male diagnosed with mucopolysaccharidosis type III. He developed AIHA + 144 days after umbilical cord blood stem cell transplantation. DIAGNOSES: Genetic testing, cranial CT, blood cell analysis, direct/indirect antihuman globulin test.

interventionsTreatment encompasses red blood cell transfusion to rectify anemia, intravenous administration of methylprednisolone, calcium gluconate, alkaline solutions, plasma exchange, rituximab, gamma globulin, and more symptomatic therapies. The symptoms of hemolysis were inadequately managed throughout the disease progression and were alleviated upon therapy with daratumumab. OUTCOMES: The treatment was conducted without adverse effects, and the problem was well managed. A telephonic follow-up with the patient was conducted by our department 546 days post-discharge. The child's cognitive ability, linguistic skills, and other neurological processes remained stable without further decline. LESSONS: This report discusses a child with MPS III, a rare disorder characterized by complex and varied phenotypes, including distinctive facial features, short stature, cognitive regression, varying degrees of skeletal deformities, and hepatosplenomegaly. The patient has autoimmune hemolysis after umbilical cord blood transplantation. For patients who do not respond to rituximab and gamma globulin, daratumumab may be considered.