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60 Plasma KCE Reports 120 bleeding occurrence. In some countries, IG is used as the first line treatment (e.g. France) and in other countries as a second line or as one of the treatment options. • In adults: Adult ITP usually has an insidious onset and is typically a chronic disease. Its incidence increases with age. IG is usually recommended in cases with risk of serious bleeding, as part of a multi-modal therapy. Other treatments include intravenous steroids, anti-D immune globulin, vincristine, platelet transfusions and factor VIIa. However, recommendations for chronic ITP widely vary per guideline. A systematic literature review was published in 2008 as part of a health technology assessment undertaken by the Canadian drug agency. 62 It included 28 RCT: 15 RCT among children with acute ITP, 4 RCT among children with chronic disease, 7 RCT among adult ITP and 2 RCT among mixed populations. IG was compared with steroids, anti-D immunoglobulin, modified or different doses of IVIG, or close observation. The review only included English language publications. • In children: In one trial that compared IG with close observation, IG was associated with earlier improvements in platelet counts. IG also showed higher effectiveness compared with steroids for early recovery of thrombocytopenia in acute ITP. The advantage of IG over anti-D immunoglobulin is uncertain due to sparse evidence. The optimal dose of IG has not been established. There was insufficient evidence to determine if specific subgroups of children with acute ITP may receive preferential benefit from IG treatment. The role of IG in chronic childhood ITP could not be established, because evidence from scant, poorly designed, and poorly reported RCT could not be synthesized. • In adults: Two RCT compared IG with steroids in adults with newly diagnosed ITP: in one, no advantage for IG was found; in the other study, IG was more effective than steroids for the short-term improvement of thrombocytopenia and gave a more rapid and sustained response. 63 IG was associated with more severe adverse events that prolonged hospitalization. The review concluded that sparse evidence indicates that IG may be more efficacious than high dose corticosteroids in improving platelet counts in the short term, though its effect on clinical outcomes remains indeterminate. Limited evidence could not demonstrate the superiority of a 2 g/kg dose of IVIG over a 1 g/kg dose; a dose of 1 g/kg, however, may be better than a dose of 0.5 g/kg. For the long-term management of adult ITP, data were insufficient and studies were heterogeneous, preventing any conclusion on whether IG has an advantage over other treatments. A US study compared the cost-effectiveness of IG, anti-D, methylprednisolone and prednisone. 64 Therapies with IG and methylprednisolone were less effective and more expensive than anti-D and prednisone, respectively. However, this analysis has been debated by many authors, as it is based on several questionable assumptions; for instance, they assumed that all ITP patients received treatment, all children responded 65, 66 to the initial management strategy, and IG is not more effective than steroids. A more recent Chinese systematic review of studies published between 1990 and 2007 showed that the use of steroids provided additional life years and was more costeffective compared with IG, but that transferability of results to other countries was low. 62
KCE Reports 120 Plasma 61 • In children with acute ITP, evidence suggests that IG improves more rapidly platelet counts than no treatment or steroids. The role of IG in chronic childhood ITP could not be established. • In adult ITP, sparse evidence indicates that IG may be more efficacious than steroids in improving platelet counts in the short term, though its effect on clinical outcomes is unclear. For long term treatment of chronic ITP, studies did not allow to determine the benefit of IG and its advantage over other treatments. Fetal/Neonatal allo-immune thrombocytopenia Fetal or neonatal allo-immune thrombocytopenia (FNAIT) accounts for 27% of cases of severe foetal and neonatal thrombocytopenia and is the most frequent cause of foetal/neonatal intracranial haemorrhage. 67 FNAIT occurs when the mother produces antibodies against fetal platelet-specific antigens that the foetus has inherited from the father. These antibodies may easily cross the placenta as early as the 14th week of gestation and cause fetal thrombocytopenia. Transfer of antibodies increases as gestation progresses, until a maximum level is attained in the late third trimester. Its incidence is estimated to be one in 1000-2000 births. The recommended antenatal therapy, aiming at preventing intracranial haemorrhage, mainly consists of maternal infusions of IG, with or without steroids, or intrauterine transfusion of antigen compatible platelets. 68 Steroids are often used in the management of refractory cases. A Cochrane review published in 2005 identified one RCT involving 54 pregnant women. However, this trial only compared intravenous IG plus steroids with intravenous IG alone, thus not covering the research question. 68 A systematic review published after the Cochrane search period identified an additional RCT, published in 2007, comparing again the effectiveness of IG plus prednisone to IG alone in 73 women. 69 No RCT comparing IG to placebo has been found, likely because IG is currently the first line treatment in most settings. However, 26 observational studies on IG efficacy have suggested an improvement in clinical outcome and probable reduction in risk for intracranial haemorrhage. Though no meta-analysis wad performed and in spite of the drawbacks of observational studies, most of current practice is based in their findings. 68 Though no RCT have been conducted to study the IG effectiveness in fetal/feonatal allo-immune thrombocytopenia, data from observational studies suggested a benefit of IG. Isoimmune haemolytic jaundice in neonates Both rhesus factor and ABO incompatibilities between maternal and fetal or neonatal cells can result in autoimmune haemolytic disease of the new-born. The use of anti-D prophylaxis in rhesus negative women has led to a marked decline in haemolytic disease of the newborn. However, anti-D immunoglobulin does not always prevent sensitization as a high proportion of haemolytic disease of the newborn is caused by antibodies to antigens other than D. 70 Exchange transfusion and phototherapy have traditionally been used to treat affected newborn infants. However, exchange transfusion is related to increased mortality and morbidity. New therapies, aiming at reducing the need for exchange transfusion, include IG. A Cochrane review published in 2002 included three RCT, involving 189 term and preterm infants with rhesus and ABO incompatibility. The use of exchange transfusion decreased significantly in the IG treated group, with a lower mean number of exchange transfusions per infant. None of the studies assessed long term outcomes. Authors considered that the applicability of the results is limited. A more recent systematic review published in 2003 included 4 RCT to compare neonatal IG plus phototherapy to phototherapy alone. 71 It also found that fewer infants in the IG group required exchange transfusion. The IG group also required lower duration of phototherapy and shorter hospital stays than the control group. In isoimmune haemolytic disease in infants, IG reduced the need for exchange transfusion and phototherapy and lead to shorter hospital stays.
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KCE Reports 120B Plasma i PRÉFACE
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KCE Reports 120B Plasma iii MÉTHOD
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KCE Reports 120B Plasma v En ce qui
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KCE Reports 120 Plasma 3 GLOSSARY A
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KCE Reports 120 Plasma 121 APPENDIX
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