Intracranial Hemorrhage in Children With Inherited Bleeding Disorders: A Single Center Study in China.

BACKGROUND: Intracranial hemorrhage (ICH) is a life-threatening condition in children. Inherited bleeding disorders (IBD) have high risk of ICH. AIM: This single center study aims to identify the incidence, risk factors, and neurological outcome of ICH in children who suffer from IBD. METHODS: From 2005 to 2017, 241 children with IBDs from Nanfang hospital, Department of Pediatrics, were evaluated. The ICH episodes were identified by medical history, general physical examination, detailed neurological examination, and computed tomographic or magnetic resonance imaging examination. The risk factors, location of ICH, management strategies, and outcome were noted. RESULTS: ICH was confirmed in 54/241 (22.4%) children with IBD among them 52/54 (96.2%) (95% confidence interval [CI], 91.1%-99.9%) were hemophilia A and hemophilia B patients. The overall risk of ICH among children with IBD was 22.4% (95% CI, 17.2%-27.8%). The median age of ICH was 30 months (0 to 204) and 18/54 (33.3%) (95% CI, 20.3%-46.3%) children had an ICH in the first year of life. Twenty-eight of 52 (53.8%) hemophilic children with ICH were assessed for inhibitor of FVIII and FIX. Nine of 28 (32%) hemophilic children with inhibitor developed the ICH. Six of 52 (11.5%) (95% CI, 2.6%-20.5%) hemophilic children had multiple episodes of ICH in which 4 were inhibitor positive. Thirteen of 54 (24%) (95% CI, 12.3%-35.9%) had positive family history of IBD. Twenty-two (36%) (95% CI, 23.7%-48.5%) of 61 ICH episodes were caused by trauma and 39 (63.9%) (95% CI, 51.5%-76.3%) were nontrauma related. Subdural hematoma was most frequently observed. Mortality risk from ICH in children with IBD was 5/54 (9.2%) (95% CI, 1.3%-17.2%). Eleven (22.4%) (95% CI, 10.3%-34.6%) of 49 survivors had known neurological squeal, whereas 38 (77.5%) (95% CI, 65.4%-89.7%) had no documented evidence of neurological impairment. CONCLUSIONS: Hemophilia is the most common IBD and most frequently associated with ICH. Risk and consequences of ICH in IBD were high during the first year of life while in older children better outcome may be expected. The optimal management of ICH depends on immediate recognition and prompt replacement therapy.

Transplant Outcomes in Beta-Thalassemia Major Patients Receiving Combined Granulocyte Colony-Stimulating Factor-Primed Bone Marrow and Cord Blood Graft Compared to Granulocyte Colony-Stimulating Factor-Primed Bone Marrow Alone.

BACKGROUND: Hematopoietic stem cell transplantation (HSCT) is currently the only curative treatment for thalassemia majorTM. Graft rejection (GR) and graft-versus-host disease (GVHD) are the primary obstacles to a successful outcome. METHODS: We conducted a retrospective study of HSCT in 29 children (median age at transplantation: 6 years) with Beta-thalassemia (ß-TM) after the combined infusion of granulocyte colony-stimulating factor-primed bone marrow (G-BM) and cord blood (CB) from the human leukocyte antigen (HLA)-identical sibling donors. We also compared the outcomes of the co-transplanted children with those of children with ß-TM who received G-BM alone from an HLA-identical sibling donor (n = 26). RESULTS: Compared to the G-BM transplant (G-BMT) recipients, those who received a co-transplant had a lower incidence of grade ≥II acute (17.24 vs. 30.7%, p = 0.047) and limited chronic (0 vs.15.4%, p = 0.022) GVHD as well as a lower incidence of GR (0 vs. 7.7%, p = 0.132). Neutrophil recovery time was faster in the co-transplant group (18.5 vs. 21 days, p = 0.04). All the patients were monitored until December 31, 2016; the median follow-up time was 74 months, and the 5-year thalassemia-free survival rate was 89.7% in the co-transplant group and 84.6% in the G-BMT-alone group (p = 0.590). CONCLUSIONS: A combined CB and G-BM graft from an HLA-identical sibling donor is an effective treatment option for TM in children, with less acute and chronic GVHD.