The IX t h Makassed Medical Congress - American University of Beirut

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T h e I X t h M a k a s e d M e d i c a l C o n g r e s s ADVANCES IN PEDIATRIC SICKLE CELL DISEASE Miguel R. Abboud MD Sickle cell disease is an inherited hemoglobinopathy affecting over 80,000 people in the US and many more worldwide including the Arab world. The myriad clinical complications of sickle cell disease have their starting point in a single amino acid substitution in the b chain of human hemoglobin. This substitution results is in sickle hemoglobin (Hb S) that polymerizes under conditions such as hypoxemia and acidosis. The polymerization causes perturbations in the erythrocyte integrity which lead to hemolysis and vaso-occlusion. Recently, two clinical phenotypes of sickle cell disease have been described. The hemolysis associated phenotype is characterized by severe anemia, leg ulcers and pulmonary hypertension. When vaso-occlusion predominates the clinical picture is defined by severe painful episodes, acute chests syndrome, splenic infarction, stroke and avascular necrosis of target joints. The disease is also characterized by endothelial dysfunction and a vasculopathy. The course of the disease is very unpredictable but for many patients is associated with significant morbidity, decreased life expectancy and poor quality of life. The risk of early mortality is highest in older patients with a history of severe complication such a recurrent acute chest syndrome, renal failure and pulmonary hypertension. Chronic transfusions are effective in preventing many complications of sickle cell disease but this modality of therapy has significant complications such as iron overload and alloimunization. Hydroxyurea has been show to be very effective in ameliorating certain manifestations of the disease, such as painful crises and acute chest syndrome. This modality of therapy also and leads to a longer life expectancy. Many patients however will have poor response to hydroxyurea and still require other forms of therapy. Like for thalassemia the only curative modality is correction of the genetic defect by allogeneic hematopoietic cell transplantation. This presentation will focus on recent findings in the pathophysiology and therapy of sickle cell disease. ITP IN CHILDREN : AN UPDATE Françoise Mazingue MD ITP is frequent in young children, characterised by severe isolated thrombocytopenia and presence of auto antibodies to platelets. The risk of severe bleeding is often over estimated and seriously impacts the way of life of the child and his family, more than the disease itself would do. Most of ITP are post infectious (viral or post vaccination). Since a few years, ITP management has been modified. Bone marrow examination is usually not necessary for diagnosis if clinical examination and blood count exclude leukemia. Treatment is based on Buchanan score and platelets count. In case of mild bleeding and platelets >10 000/mm3 one can just clinically survey, hospitalization is not necessary. On the other hand, in case of severe hemorrhage or life threatening bleeding, treatment will associate IV Ig to steroids (4mg/kg, during 4 days), sometimes platelets transfusions are necessary. In case of refractory ITP, one can have to use Vincristine and sometimes Rituximab. Chronic ITP is lasting for more than 12 months. The diagnosis of primary chronic ITP remains one of exclusion. There are a lot of secondary ITP. It’s necessary to establish diagnosis with accuracy to adapt 56
treatment. Specific treatment of these diseases can improve thrombocytopenia, for example hydroxylchloroquine in lupus or Rituximab in lupus or Evans syndrome. In case of primary chronic ITP, there is no more place for prolonged corticotherapy. A treatment will be initiated only if there are mucosis bleedings or headach in case of intracranial bleeding. Splenectomy is still indicated in chronic refractory ITP, It can cure ITP in 60-70% cases .A prevention of infections by encapsulated agents is necessary: vaccination and antibiotics prophylaxy Some studies with Thrombopoietin analogs are going to be initiated in children INDICATIONS OF BONE MARROW TRANSPLANT (BMT) IN CHILDREN Françoise Mazingue MD Since 30 years progress in understanding and treatment of hematological diseases have been important. A lot of children can be cured. BMT has a full place in the treatment of malignant hemopathies but it also takes an important place in the treatment of non malignant hemopathies, solid tumors and in the treatment of some rare diseases such as metabolic diseases, congenital BM dysfunctions and severe Immunodeficiencies - BMT and malignant hemopathies: - Auto BMT keeps a few indications in the treatment of Hodgkin disease and in non Hodgkin lymphomas. In theses diseases, BM is usually normal and BMT just allows an intensification of chemotherapy, shortening aplasia, with less infectious complications. Treatment of the disease doesn’t consist in BMT but in conditioning chemotherapy. - Allo BMT is the most frequent situation Its interest is double: - First normal BM of the donor takes the place of leukemic BM, - Secondly, alloreactivity and GVL act against leukemia and can evitate relapse. Indications of allo BMT are essentially: - leukaemia in first remission with bad prognosis factors - leukaemia in second remission after a relapse - preleukemic syndromes , myelodysplasia before leukemic transformation BMT and non malignant diseases: In that case, geno identical donors are prefered, because GVH and GVL are not usefull Severe idiopathic aplasia: BMT must be done as soon as possible if there is a good donor (geno identical) Congenital bone marrow dysfunctions such as Fanconi anemia, pure red cell aplasia refractory to steroids, congenital amegacaryocytosis, severe congenital neutropenia, refractory to GCSF…. 57
Page 1 and 2:
The IX th Makassed Medical Congress
Page 3 and 4:
CONGRESS PRESIDENT Said Sayegh, MD,
Page 5 and 6: GENERAL INFORMATION
Page 7 and 8: • Target Audience: Physicians, Pr
Page 9 and 10: Durriya Sinno, MD Pediatrics Americ
Page 11 and 12: Moderators
Page 13 and 14: PEDIATRICS Bassem Abou Merhi, MD Pe
Page 15 and 16: Maroun Sokhn, MD Pediatrics Saint G
Page 17 and 18: Scientific Program
Page 19 and 20: Session 3: Intensive Care Neonatolo
Page 21 and 22: Session 1: Pediatric Intensive Care
Page 23 and 24: 8:00 - 8:05 08:05 - 08:10 Welcome r
Page 25 and 26: Cardiology Session III: Interventio
Page 27 and 28: Cardiology Session VI: Intervention
Page 29: Cardiology Symposia (NON-CME) Frida
Page 32 and 33: T h e I X t h M a k a s e d M e d i
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The IX t h Makassed Medical Congress - American University of Beirut

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