Efficacy and tolerability of old and new drugs used in the treatment of immune thrombocytopenia: Results from a long-term observation in clinical practice

PLoS One. 2018 Jun 1;13(6):e0198184. doi: 10.1371/journal.pone.0198184. eCollection 2018.

Abstract

Background: Many patients with immune thrombocytopenia (ITP) may require special attention and long-term treatment. Little is known on the efficacy and tolerability of the drugs used in practice.

Material and methods: We retrospectively reviewed the results of therapy of 400 patients with chronic ITP. All Patients were treated at our institution between 1996-2016 under consideration of guidelines, general recommendations, and individual aspects, including gender, age, weight, comorbidity, patient's medical history and bleeding risk.

Results: Treatment was not required in 25% of patients (n = 100) during observation. In treated patients (n = 300), the rate of patients that responded and tolerated treatment with prednisolone was 59% (52/88), with azathioprine 32% (29/90), with eltrombopag 49% (31/63), with romiplostim 59% 27/45, with IVIG (intravenous immunoglobulines) 75% (94/126), with anti-D 37% (19/52) and with dexamethasone 60% (25/42) patients. Eighteen treated patients (6%) entered sustained remission after treatment with various drugs. Twenty-six patients underwent splenectomy (Splx) resulting in sustained remission in 15 cases (60%). Only two patients remained refractory to Splx and to all used drugs.

Discussion: None of the currently available drugs used in the treatment of ITP are invariably safe and effective. Responses, the duration of response, intolerability, and the course of disease are unpredictable. Although the treatment of ITP has considerably improved in the recent years, the currently available drugs may rarely cure affected patients. The need for safe and effective therapy in ITP is evident. Optimal treatment decisions for each patient remains a challenge in many cases.

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Azathioprine / adverse effects
  • Azathioprine / therapeutic use
  • Benzoates / adverse effects
  • Benzoates / therapeutic use
  • Child
  • Child, Preschool
  • Combined Modality Therapy
  • Cyclosporine / adverse effects
  • Cyclosporine / therapeutic use
  • Dapsone / adverse effects
  • Dapsone / therapeutic use
  • Dexamethasone / adverse effects
  • Dexamethasone / therapeutic use
  • Drug Resistance
  • Drug Therapy, Combination
  • Female
  • Humans
  • Hydrazines / adverse effects
  • Hydrazines / therapeutic use
  • Immunoglobulins, Intravenous / adverse effects
  • Immunoglobulins, Intravenous / therapeutic use
  • Immunosuppressive Agents / adverse effects
  • Immunosuppressive Agents / therapeutic use*
  • Male
  • Middle Aged
  • Prednisolone / adverse effects
  • Prednisolone / therapeutic use
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Purpura, Thrombocytopenic, Idiopathic / surgery
  • Purpura, Thrombocytopenic, Idiopathic / therapy
  • Pyrazoles / adverse effects
  • Pyrazoles / therapeutic use
  • Receptors, Fc / therapeutic use
  • Recombinant Fusion Proteins / adverse effects
  • Recombinant Fusion Proteins / therapeutic use
  • Remission Induction
  • Retrospective Studies
  • Rho(D) Immune Globulin / adverse effects
  • Rho(D) Immune Globulin / therapeutic use
  • Rituximab / adverse effects
  • Rituximab / therapeutic use
  • Splenectomy
  • Thrombopoietin / adverse effects
  • Thrombopoietin / therapeutic use
  • Treatment Outcome
  • Young Adult

Substances

  • Benzoates
  • Hydrazines
  • Immunoglobulins, Intravenous
  • Immunosuppressive Agents
  • Pyrazoles
  • RHO(D) antibody
  • Receptors, Fc
  • Recombinant Fusion Proteins
  • Rho(D) Immune Globulin
  • Rituximab
  • Dexamethasone
  • Cyclosporine
  • Dapsone
  • Thrombopoietin
  • Prednisolone
  • romiplostim
  • Azathioprine
  • eltrombopag

Grants and funding

The authors received no specific funding for this work.