Article Text

Download PDFPDF
CP-138 Successful off-label use of eltrompobag in pregnant woman with congenital thrombopenia
  1. J Raymond1,
  2. J Descout1,
  3. A Aouba2,
  4. S Barbault-Foucher1,
  5. A Rieutord1,
  6. A Decottignies1
  1. 1Hôpital Antoine Béclère- APHP, Pharmacie, Clamart, France
  2. 2Hôpital Antoine Béclère- APHP, Médecine Interne, Clamart, France


Background Congenital thrombopenia is a rare disease. We report the case of a pregnant 25-year-old woman, suffering from a new form of severe congenital thrombopenia due to an autosomal recessive mutation in the PRKACG gene, found in 2014. The patient was advised not to plan such a high-risk pregnancy. A medical termination of pregnancy was rejected by the couple.

Purpose To report the off-label use of eltrombopag in a pregnant woman with congenital thrombopenia, allowing reaching a suitable platelet count for a safe delivery (vaginal birth: 30–50 G/L, caesarean: 50 G/L), scheduled at 35–37 weeks of amenorrhea (WA).

Material and methods Several off-label drugs were successively tested: intravenous immunoglobulin G (1 g/kg/day), and two thrombopoietin receptor agonists, romiplostim by the subcutaneous route (250 µg/week) and eltrombopag by oral route (50 to 125 mg daily). A multidisciplinary committee including haematologists, internists, obstetricians, pharmacists and pharmacologists from the Centre for Teratogenic Agents took the decision based on a risk-benefit approach. Twice-monthly platelet counts were performed and the dose was adjusted accordingly.

Results Intravenous immunoglobulin G showed an initial efficiency, raising the platelet count up to 115 G/L, but a relapse was observed after 1 month. This treatment was replaced by romiplostim, 6 months before pregnancy. Romiplostim stabilised the platelet count (275 G/L) until 6 WA and then lost progressively its effectiveness. The change to eltrombopag occurred at 20 WA. Platelet count increased up to 335 G/L at 24 WA and decreased gradually while keeping enough platelets until delivery. The patient gave birth by caesarean at 35 WA and 2 days, without haemorrhagic complications and platelet count was 80 G/L. The newborn was not affected by the disease.

Conclusion Eltrombopag was the most effective treatment in the management of this patient’s thrombopenia and it enabled a high-risk pregnancy to achieve a successful outcome.

References and/or Acknowledgements No conflict of interest.

Statistics from

Request Permissions

If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.