Objective To assess feasibility and safety of providing autologous umbilical cord blood (UCB) cells to neonates with hypoxic-ischemic encephalopathy (HIE). Study design We enrolled infants in the Intensive Care Nursery who were cooled for HIE and had available UCB in an open-label study of non-cyropreserved autologous volume- and red blood cell-reduced UCB cells (up to four doses adjusted for volume and RBC content,1 – 5 × 107cells/dose). We recorded UCB collection and cell infusion characteristics, and pre- and post- infusion vital signs. As exploratory analyses we compared cell recipients’ hospital outcomes (mortality, oral feeds at discharge) and one year survival with Bayley III scores ≥ 85 in 3 domains (cognitive, language, and motor development) with cooled infants who did not have available cells. Results Twenty-three infants were cooled and received cells. Median collection and infusion volumes were 36 and 4.3 milliliters. Vital signs including oxygen saturation were similar before and after infusions in the first 48 postnatal hours. Cell recipients and concurrent cooled infants had similar hospital outcomes. Thirteen of 18 (74%) cell recipients and 19 of 46 (41%) concurrent cooled infants with known 1 year outcomes survived with scores ≥ 85. Conclusions Collection, preparation and infusion of fresh autologous UCB cells for use in infants with HIE is feasible. A randomized double-blind study is needed.

Joint haemorrhage and subsequent haemophilic arthropathy are significant complications of haemophilia. The pathophysiology involves inflammation and angiogenesis. Cyclo-oxygenase 2 (COX-2) inhibitors have anti-inflammatory, anti-angiogenic and analgesic properties yet do not affect platelet function in the manner of traditional non-steroidal anti-inflammatory drugs. We have previously reported the successful use of rofecoxib in the management of haemophilic arthropathy and currently report our use of celecoxib. A retrospective chart review was conducted of all patients with haemophilia A or B seen at the Children's Hospital of Orange County and treated with celecoxib for chronic synovitis, target joint or pain. Efficacy in chronic synovitis and pain reduction was judged subjectively as effective, partially effective or ineffective. Efficacy in resolution of target joints was judged as effective if the target joint resolved or ineffective if it did not resolve. Twelve patients between 9 and 54 years old were treated for a total of 14 courses of celecoxib treatment. All courses were evaluated for safety and efficacy. Celecoxib was used in eight patients with chronic synovitis, three patients with pain, and in one patient with a target joint on three occasions. A response was noted in seven of eight for synovitis, three of three with pain, but no response in the target joint patient. No serious adverse events including hypertension were observed. This is the first study evaluating celecoxib as adjunctive therapy in haemophilia and suggests that celecoxib is safe and effective in treating chronic synovitis and joint pain similar to the previous study of rofecoxib.

Higher Mg dose was associated with higher SIP and death risk among infants with the lowest birthweights. Validation of this observation in larger populations is warranted.

Joint haemorrhage and subsequent haemophilic arthropathy are significant complications in haemophilia. The pathophysiology involves inflammation and angiogenesis. Cyclooxygenase-2 (COX-2) inhibitors are anti-inflammatory agents, which have potent anti-inflammatory, anti-angiogenic and analgesic properties yet do not affect platelet function in the manner of traditional non-steroidal anti-inflammatory drugs. These properties make such agents potentially useful as adjunctive therapy in haemophilia. There is only one prior report describing rofecoxib treatment in a single haemophilia patient. Our objectives were to determine the safety and efficacy of rofecoxib in treating acute haemarthrosis, chronic synovitis, target joints and pain. We conducted a retrospective medical record review of patients treated with rofecoxib for acute haemarthrosis, chronic synovitis, target joint or pain. The safety and efficacy of rofecoxib treatment were determined based on subjective patient reports and physical examinations during follow-up clinic visits. A total of 28 patients between 3 and 37 years of age were treated for a total of 42 courses of rofecoxib treatment. All courses were evaluated for safety and 31 for efficacy. Rofecoxib was used for eight acute haemarthrosis, four target joints, seven cases of synovitis and 12 episodes of pain. Efficacy was demonstrated particularly for chronic synovitis and pain and no serious adverse events occurred. This is the largest study to date evaluating COX-2 inhibitors as adjunctive therapy in haemophilia and suggests that these agents may be an important adjunctive therapy in the management of haemophilia.