High-dose methylprednisolone for veno-occlusive disease of the liver in pediatric hematopoietic stem cell transplantation recipients

摘要

Pediatric Hematology Oncology Journal 2 (2017) S9eS31gastrointestinal tract (GIT). Although PI is a common finding in neonatal necrotizing enterocolitis, it is otherwise rare in paediatric patients. Cases of PI have been described in children with obstructive lesions of the GIT, chronic obstructive pulmonary disease, collagen vascular diseases, acute leukaemia, congenital immunodeficiency and acquired immunocompro- mised states. Recently, PI has been reported after bone marrow trans- plantation (BMT) in adults. The development of PI in children following allogeneic BMT has not been previously described. Our objective here is to report the PI after allogeneic BMT in paediatrics patient and discuss the factors that contribute to the development of PI in these patients. Methods: Two children, aged 1year [a case of Wiskott Aldrich Syndrome (WAS)] and 6 years [a case of relapsed acute lymphoblastic leukaemia] developed PI at day 52 and 41 after transplant and was associated with abdominal symptoms and clinical signs. Both patients had gut graft- versus-host disease (GVHD) at some time of PI and were receiving corti- costeroids. Both patients were treated with supportive care and broad- spectrum antibiotics, and PI resolved in 15 days and 7 days after onset. Results: Both patients recovered from PI. Eventually one patient died of gram negative sepsis and other is well 16 months post-transplant. Conclusion: PI can be one of the manifestation of gut GVHD. SCT-1.3 ROLE OF G-CSF PRIMING OF DONOR MARROW PRIOR TO TRANSPLANT IN LOW RISK HLA MATCHED SIBLING TRANSPLANT IN CHILDREN WITH SEVERE THALASSEMIA Stalin Ramprakash a,*, Rajat Agarwal b, Rakesh Dhanya b, Priya Marwah c, Rajpreet Soni c, Naila Yaqub d, Itrat Fatima d, Lallindra Gooneratne e, Senani Williams f, Sadaf Khalid g, Santanu Sen h, Lawrence Faulkner a,i. a Sankalp-People Tree Centre for Pediatric Bone Marrow Transplantation, People Tree Hospitals, Bangalore, India; b Sankalp India Foundation, India; cBone Marrow Transplantation Unit, South East Asia Institute for Thalassemia, Jaipur, India; dBone Marrow Transplantation Unit, Children's Hospital Pakistan Institute of Medical Sciences, Islamabad, Pakistan; eBone Marrow Transplantation Unit, Central Asiri Hospital, Sri Lanka; fBone Marrow Transplantation Unit, Nawaloka Hospital, Colombo, Sri Lanka; gBoard, Cure2Children Foundation, Islamabad, Pakistan; hKokilaben Dhirubhai Ambani Hospital, Mumbai, India; iCure2Children Foundation, Italy * Background and objective: Veno-occlusive disease (VOD) of the liver is a well-recognized serious complication of hematopoietic stem cell transplantation (HSCT), with few successful treatment modalities available. Use of Defibriotide has shown some response in a few patients. Some reports have also demonstrated successful use of high dose steroids in adults, but experience in the paediatric population is lacking. In a country like ours procuring Defibriotide timely and the cost involved are the major drawback which increases the mortality in pediatric HSCT recipients who develop severe VOD. Our objective is to evaluate the efficacy of the high-dose Methylprednisolone in VOD of the liver.