Combining thrombopoietin receptor agonists with immunosuppressive drugs in adult patients with multirefractory immune thrombocytopenia, an update on the French experience

Br J Haematol. 2023 Aug;202(4):883-889. doi: 10.1111/bjh.18893. Epub 2023 May 29.

Abstract

Combining drugs could be an effective option for treating multirefractory ITP, that is, patients not responding to rituximab, thrombopoietin receptor agonists (TPO-RA) and splenectomy. We conducted a retrospective, multicenter, observational study including multirefractory ITP patients who received a combination of a TPO-RA and an immunosuppressive drug. We included 39 patients (67% women, median age 59 years [range 21-96]), with a median ITP duration of 57 months [3-393] and a median platelet count at initiation of 10 × 109 /L [1-35]. The combination regimen was given for a median duration of 12 months [1-103] and included eltrombopag (51%) or romiplostim (49%), associated with mycophenolate mofetil (54%), azathioprine (36%), cyclophosphamide (5%), cyclosporin (3%) or everolimus (3%). Overall, 30 patients (77%) achieved at least a response (platelet count ≥30 × 109 /L and at least doubling baseline during at least 3 months), including 24 complete responses (platelet count >100 × 109 /L during at least 3 months) with a median time to response of 30 days [7-270] and a median duration of response of 15 months [4-63]. Severe adverse event related to ITP treatment was observed in 31%. In conclusion, this study confirms that some patients with multirefractory ITP can achieve long lasting response with this combination.

Keywords: ITP; TPO-RA; combination therapy; immunosuppressive drug.

Publication types

  • Observational Study
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Benzoates / therapeutic use
  • Female
  • Humans
  • Hydrazines / adverse effects
  • Male
  • Middle Aged
  • Platelet Count
  • Purpura, Thrombocytopenic, Idiopathic* / chemically induced
  • Purpura, Thrombocytopenic, Idiopathic* / drug therapy
  • Receptors, Fc / therapeutic use
  • Receptors, Thrombopoietin / agonists
  • Recombinant Fusion Proteins / adverse effects
  • Retrospective Studies
  • Rituximab / adverse effects
  • Thrombopoietin / adverse effects
  • Young Adult

Substances

  • Receptors, Thrombopoietin
  • Rituximab
  • Receptors, Fc
  • Thrombopoietin
  • Benzoates
  • Hydrazines
  • Recombinant Fusion Proteins