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Clinical determinants as indication for the treatment of ITP patients. Marc MICHEL M.D Henri Mondor University Hospital Assistance-publique Hôpitaux de Paris Créteil, France. Questions. Who should be treated ? When ? How ?. Risk of ICH and mortality rate in ITP: adults.
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Clinical determinants as indication for the treatment of ITP patients Marc MICHEL M.D Henri Mondor University Hospital Assistance-publique Hôpitaux de Paris Créteil, France
Questions • Who should be treated ? • When ? • How ?
Risk of ICH and mortality rate in adult’s ITP: risk factors ? • Causes of deaths: Infections are at least as frequent as hemorrhage • Patients with chronic refractory ITP and/or previous hemorrhagic events and a plt ct < 30 x 109/L have the highest risk of bleeding • Elderly patients are at high risk of severe bleeding* (ICH) ? => controversial (> 60 > 70 ?....) *Cortelazzo S. Blood 1991; 77:31
Risk of ICH and mortality rate in ITP: children Kühne T et al. J Pediatr. 2003; 143:605 • N = 2540 children (< 16 years) with ITP (ICIS registry) • Mean initial plt ct ~ 15x109/L (< 20x 109/L in 75%) • N = 3/1742 patients with a f.u of at least 6 months had a ICH (0.17%) 1of whom died (mortality rate = 0.05%) • Mortality rate of ICH in children with ITP ~ 55%* *Butros LJ. J Ped Hem Onc 2003;25:660
Risk of ICH and mortality rate in ITP • In a large majority of patients, ITP has a benign course and a low mortality rate • An active therapeutic approach must be therefore considered only: • Acute phase => in patients with a plt ct < 20-30 x 109/L • Chronic ITP => in patients with chronic ITP and bleeding manifestations
Other determinants for the treatment of ITP ? • In preparation for surgery or any invasive procedure (Guidelines* but no strong evidence) • In patients with a plt ct < 50 x 109/L who need to be treated with anticoagulants, or aspirin and/or clopridrogel • Before delivery (threshold = 50 to 70 x 109/L) *BCSH guidelines Br J Haematol 2003; 120: 574
Treatment of adult’s ITP Acute/initial phase* 1) IVIg is more rapidly and more frequently effective than high dose of steroids (HDMP) 2) A short course (x 3 weeks) of oral prednisone required after IVIg infusion BUT HDMP lead to an immediate response in 60% of the cases IVIg is much more expensive than steroids +++ *Godeau B. Lancet 2002;359:23
A therapeutic strategy based on a bleeding score (7 sites) * Only the highest score is taken into consideration
4 5 + 9 =
Therapeutical strategy based on a bleeding score ITP Patients with plt ct ≤ 20.109/L Bleeding score Score ≤ 8 Score > 8 Failure Steroids alone • Platelet ct < 20.109/L on day 3 • Worsening of bleeding score Success IVIg + steroids
59 ITP patients with < 20.109/L platelets 44 patients Score ≤ 8 15 patients Score > 8
59 patients < 20.109/L 44 patients Score ≤ 8 Steroids 38 IVIg alone 6 Failure 9 Success 29 2 known resistance to steroids 1 Fever 1 severe Hypertension 1 Protocol violation 1 Diabetes IVIg 15 Patients
Results 59 patients • 30/59 (51%) patients were successfully treated by steroids alone (including 1 pt with a score > 8) • 9/38 (23%) patients with an initial score ≤ 8 did not respond to steroids • Overall 29/59 (49%) did receive IVIg (no failure)
Conclusion A therapeuthic strategy based on a bleeding score is safe and is a good « IgIV sparing strategy » (avoid the use of IVIg in 51% of the cases) Khellaf M et al. Haematologica 2005; 90:829-32
What would be an ideal bleeding score ? • Easy and rapid • Good interobserver reliability • Relevant in detecting the patients with the highest risk of life-threatening hemorrhage • Helpful to stratify the therapeutical strategy => Could be one of the objectives of the WG in the next future…