No abstract

Perioperative management of severe congenital protein C deficiency remains unestablished. This deficiency is often treated with anticoagulants, such as warfarin. Although anticoagulants need to be perioperatively discontinued, there are few methods for the management of such patients. We adopted a method for administering prothrombin complex concentrates (PCC), which includes intermittent administration of inactive protein C (PPSB-HT), and examined its outcome as a perioperative management approach for severe congenital protein C deficiency. Three patients underwent our perioperative management six times. We monitored activity levels of protein C, factor IX, and so forth. These patients could be perioperatively managed with PCC treatment.

Cold agglutinin disease (CAD) is a rare form of autoimmune hemolytic anemia (AIHA), usually caused by autoantibodies that react optimally at low temperatures. Although rituximab has been shown to be effective against adult CAD, no established treatment is available for pediatric AIHA due to its rarity in infancy and childhood. We report an infantile case of CAD that responded to intravenous immunoglobulin (IVIG) treatment. A 2-month-old previously healthy Japanese female presented with jaundice and poor feeding 3 days after receiving the first doses of Haemophilus influenzae type b conjugate vaccine and pneumococcal conjugate vaccine. No exposure to cold temperature could be identified. There was no family history of anemia, spherocytosis, or splenectomy. Physical examination revealed scleral icterus and generalized pallor, but no acrocyanosis or hepatosplenomegaly. Her laboratory data showed a hemoglobin (Hb) level of 3.8 g/dL, mean corpuscular volume of 87.3 fl, reticulocyte count of 3.4%, total bilirubin (TB) of 10.4 mg/dL, direct bilirubin of 0.8 mg/dL, lactate dehydrogenase (LDH) of 606 U/l, haptoglobin of 3 mg/dL, and no hemoglobinuria. A direct antiglobulin test (DAT) with polyspecific anti-IgG and anti-C3 was positive at 4°C and room temperature but negative at 37°C. Peripheral blood smears revealed no spherocytes. Bone marrow smears showed erythroid hyperplasia. Based on clinical signs and laboratory evaluations, we diagnosed her with cold AIHA. Further investigation concluded that monospecific DAT was positive for IgG and negative for C3. Her cold agglutinin titer was 1:16. The autoantibody was specific for the I antigen. The Donath-Landsteiner antibody test was negative, ruling out paroxysmal cold hemoglobinuria. Infectious agents were excluded with serology tests. These laboratory findings led to a diagnosis of low-titer CAD.

In 2019, defibrotide (DF) was approved for use to treat sinusoidal obstruction syndrome in Japan. However, few studies in the literature report on its use in children. We retrospectively analyzed cases of pediatric patients who underwent hematopoietic cell transplantation and developed sinusoidal obstruction syndrome, which was treated with DF in our center. Four patients with myelodysplastic syndrome(n＝1) , X-linked inhibitor of apoptosis protein(XIAP)deficiency (n＝1) , neuroblastoma(n＝1) , and acute lymphoblastic leukemia(n＝1)were included in the study. Total bilirubin levels decreased after the start of DF therapy in all cases, except in the patient with acute lymphoblastic leukemia, who died immediately after starting DF therapy owing to worsening of the primary disease. Of the three patients whose total bilirubin levels decreased, two recovered and one died because of multiple organ failure. Apparent adverse events with DF administration were not observed, and a decrease in total bilirubin levels early in the treatment period tended to be a predictor of treatment response. It is hoped that more reports of patients with DF administration in Japan will be accumulated in the future.