Idelalisib for treatment of B-cell malignancies

Am J Health Syst Pharm. 2016 Apr 15;73(8):547-55. doi: 10.2146/ajhp150281. Epub 2016 Mar 1.


Purpose: The pharmacology, pharmacokinetics, clinical efficacy, safety and tolerability, dosing and administration, and place in therapy of idelalisib, a targeted therapy for certain types of non-Hodgkin's lymphoma (NHL), are reviewed.

Summary: Historically, conventional cancer chemotherapy agents were recommended for the management of progressive lymphomas requiring systemic treatment; in recent years, however, emerging targeted therapies have altered the landscape of lymphoma treatment. Idelalisib, a novel oral phosphatidylinositol 3-kinase (PI3K) inhibitor, disrupts downstream signaling pathways involved in cancer cell growth and survival. Inhibition of PI3K has been demonstrated to produce durable treatment responses and improved survival outcomes in clinical trials involving patients with indolent forms of NHL. Idelalisib is indicated for use in combination with rituximab for treatment of relapsed chronic lymphocytic leukemia (CLL) and as monotherapy for relapsed small lymphocytic leukemia and follicular lymphoma after the failure of at least two systemic treatments. The recommended dosage of idelalisib is 150 mg orally twice daily; the medication can be taken without regard to mealtimes. The most common adverse effects of idelalisib include diarrhea, nausea, fatigue, cough, and pyrexia. Severe hepatotoxicity and gastrointestinal toxicities, including colitis and intestinal perforation, have also been reported in association with idelalisib use. Ongoing clinical studies are exploring the potential for expanded use of idelalisib in the management of other B-cell malignancies.

Conclusion: Idelalisib is a well-tolerated and effective treatment for patients with relapsed or refractory CLL or indolent NHL, providing a novel targeted therapeutic option for the management of these hematologic malignancies.

Publication types

  • Review

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / administration & dosage*
  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Antineoplastic Combined Chemotherapy Protocols / pharmacology
  • Humans
  • Leukemia, Lymphocytic, Chronic, B-Cell / drug therapy
  • Leukemia, Lymphocytic, Chronic, B-Cell / pathology
  • Lymphoma, Non-Hodgkin / drug therapy*
  • Lymphoma, Non-Hodgkin / pathology
  • Molecular Targeted Therapy
  • Phosphoinositide-3 Kinase Inhibitors
  • Purines / administration & dosage*
  • Purines / adverse effects
  • Purines / pharmacology
  • Quinazolinones / administration & dosage*
  • Quinazolinones / adverse effects
  • Quinazolinones / pharmacology
  • Rituximab / administration & dosage


  • Phosphoinositide-3 Kinase Inhibitors
  • Purines
  • Quinazolinones
  • Rituximab
  • idelalisib