Important Safety Information

WARNING: RISK FOR HEPATIC DECOMPENSATION IN PATIENTS WITH CHRONIC HEPATITIS C - In patients with chronic hepatitis C, PROMACTA in combination with interferon and ribavirin may increase the risk of...+

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Indication for PROMACTA® (eltrombopag)
PROMACTA is indicated for the treatment of thrombocytopenia in adult and pediatric patients 1 year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. PROMACTA should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.

Limitations of Use
PROMACTA is not indicated for the treatment of patients with myelodysplastic syndromes (MDS).

Safety and efficacy have not been established in combination with direct-acting antiviral agents used without interferon for treatment of chronic hepatitis C infection.

Resources for Your Practice

ASH & ICR Guidelines for ITP

ITP Treatment Guidelines

The American Society of Hematology (ASH) and International Consensus Report (ICR) updated their guidelines for the treatment of ITP in 2019. Key highlights for adult ITP:

  1. Short courses of steroids are recommended—no more than 6 weeks1,2
    • The ASH guidelines panel recommends a short initial course (≤6 weeks including treatment and taper) of prednisone and against a prolonged course (>6 weeks)1
    • The ICR experts state that if a response (defined as ≥50,000/mcL) is seen, predniso(lo)ne should be tapered, aiming to stop by 6 weeks—even if the platelet count drops during the taper. If no response is seen within 2 weeks, treatment should be tapered rapidly over 1 week and stopped2
  2. Shared decision making based on patient preference is important in second line1,2

In adults with ITP lasting ≥3 months who failed first-line therapy, assess patient values and preferences in regard to second-line therapy, including achieving durable response, avoiding long-term medication, and avoiding surgery.1

  • TPO-RAs:
    • The ASH guidelines suggest a TPO-RA rather than rituximab* for patients who value a durable response and want to avoid surgery. The panel specifies eltrombopag or romiplostim; this is not a classwide recommendation. Route of administration is a key consideration in the choice of TPO-RA, as individual patient preference may place a higher value on the use of an oral medication rather than an injection1
    • The ICR experts note an additional benefit of TPO-RAs: Patients responding to TPO-RAs also demonstrated improved HRQoL—with responders improving more than responders to other therapies evaluated2
  • Rituximab:
    • The ASH guidelines suggest rituximab as a second-line treatment option for patients who place a high value on avoiding long-term medication and avoiding surgery1
    • The ICR experts cite evidence from a systematic review of multiple uncontrolled trials and randomized controlled trials showing a response to rituximab in 60% of patients. Long-term durable responses occurred in 20% to 25% of adult patients2
  • Splenectomy:
    • According to the ASH guidelines panel, splenectomy should be delayed at least 1 year after diagnosis because of the potential for spontaneous remission in the first year. ASH guidelines suggest splenectomy in patients with chronic ITP (>12 months) who place a high value on achieving a durable response and avoiding long-term medication regimens1
    • The ICR experts recommend waiting ≥1 to 2 years from diagnosis before performing splenectomy because of the chance of remission or stabilization2

See the full ASH guidelines here.

See the full ICR guidelines here.

HCP Resources

Adult ITP Brochure

A resource for HCPs containing information about efficacy, dosing and administration, access, adverse events, mechanism of action, and Important Safety Information for PROMACTA® (eltrombopag)

Download the PDF here

ITP Treatment Guidelines

A resource for HCPs containing information adapted from the 2019 guidelines from the American Society of Hematology and International Consensus Report

Download the PDF here

Patient Initiation Guide

A resource for HCPs containing information on comprehensive support to help get patients started on PROMACTA based on coverage type, patient navigators, PROMACTA4U, and helpful downloadable resources for patients

Download the PDF here

Access – Sample Letters

Prior Authorization Request

Download the PDF here

Prior Authorization Denial

Download the PDF here

Prior Authorization Denial, Patient

Download the PDF here

Medical Necessity

Download the PDF here

Formulary Exception Request

Download the PDF here

Access – Appeals Kit

PROMACTA Appeals Kit

Download the PDF here

FDA, US Food and Drug Administration; HCP, health care professional; HRQoL, health-related quality of life; ITP, immune thrombocytopenia; TPO-RA, thrombopoietin receptor agonist.

*Rituximab is not FDA approved for use in ITP.3

References:

  1. Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019;3(23):3829-3866.
  2. Provan D, Arnold DM, Bussel JB, et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019;3(22):3780-3817.
  3. Rituxan [prescribing information]. South San Francisco, CA: Biogen and Genentech USA Inc; 2020.

Indication and Important Safety Information

Indication for PROMACTA® (eltrombopag)
PROMACTA is indicated for the treatment of thrombocytopenia in adult and pediatric patients 1 year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. PROMACTA should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.

Limitations of Use
PROMACTA is not indicated for the treatment of patients with myelodysplastic syndromes (MDS).

Safety and efficacy have not been established in combination with direct-acting antiviral agents used without interferon for treatment of chronic hepatitis C infection.

Important Safety Information for PROMACTA® (eltrombopag)

WARNING: RISK FOR HEPATIC DECOMPENSATION IN PATIENTS WITH CHRONIC HEPATITIS C
In patients with chronic hepatitis C, PROMACTA in combination with interferon and ribavirin may increase the risk of hepatic decompensation.

RISK OF HEPATOTOXICITY
PROMACTA may increase the risk of severe and potentially life-threatening hepatotoxicity. Monitor hepatic function and discontinue dosing as recommended.

Hepatotoxicity
PROMACTA may increase the risk of severe and potentially life-threatening hepatotoxicity.

Treatment of ITP, chronic hepatitis C, and refractory severe aplastic anemia

  • Measure serum alanine aminotransferase (ALT), aspartate aminotransferase (AST), and bilirubin prior to initiation of PROMACTA, every 2 weeks during the dose-adjustment phase, and monthly following establishment of a stable dose
  • PROMACTA inhibits UGT1A1 and OATP1B1, which may lead to indirect hyperbilirubinemia. If bilirubin is elevated, perform fractionation
  • Evaluate abnormal serum liver tests with repeat testing within 3 to 5 days. If the abnormalities are confirmed, monitor serum liver tests weekly until resolved or stabilized
  • Discontinue PROMACTA if ALT levels increase to ≥3 times the upper limit of normal in patients with normal liver function or ≥3 times baseline in patients with pretreatment elevations in transaminases and are progressively increasing; or persistent for ≥4 weeks; or accompanied by increased direct bilirubin; or accompanied by clinical symptoms of liver injury or evidence for hepatic decompensation
  • If the potential benefit for reinitiating treatment with PROMACTA outweighs the risk for hepatotoxicity, then consider cautiously reintroducing PROMACTA and measure serum liver tests weekly during the dose-adjustment phase. Hepatotoxicity may reoccur if PROMACTA is reinitiated. If liver test abnormalities persist, worsen, or recur, then permanently discontinue PROMACTA

Thrombotic/Thromboembolic Complications

  • Thrombotic/thromboembolic complications may result from increases in platelet counts with PROMACTA
  • Reported thrombotic/thromboembolic complications included both venous and arterial events, and were observed at low and at normal platelet counts
  • Portal vein thrombosis has been reported in patients with chronic liver disease receiving PROMACTA
  • To minimize the risk for thrombotic/thromboembolic complications, do not use PROMACTA in an attempt to normalize platelet counts. Follow the dose-adjustment guidelines to achieve and maintain target platelet counts

Increased Risk of Death and Progression of Myelodysplastic Syndromes (MDS) to Acute Myeloid Leukemia (AML)

  • In a clinical trial of patients with intermediate- to high-risk MDS and thrombocytopenia receiving PROMACTA, an increased number of progressions from MDS to AML and deaths have been observed compared to placebo
  • PROMACTA is not indicated for the treatment of patients with MDS

Cataracts

  • Development or worsening of cataracts with PROMACTA has been reported with a frequency of 5% to 11% in 6 clinical studies
  • Perform a baseline ocular examination prior to initiating PROMACTA. Regularly monitor patients for signs and symptoms of cataracts while on PROMACTA

Laboratory Monitoring

  • Monitor serum liver tests
  • During therapy with PROMACTA, assess complete blood counts (CBCs) with differentials, including platelet counts, weekly until a stable platelet count has been achieved. Monitor platelet counts monthly thereafter
  • Obtain CBCs with differentials, including platelet counts, weekly for at least 4 weeks following discontinuation of PROMACTA
  • When switching between the oral suspension and tablet, assess platelet counts weekly for 2 weeks, then follow standard monthly monitoring

Drug/Drug and Drug/Food Interactions

  • PROMACTA must be taken at least 2 hours before or 4 hours after any medications or products containing polyvalent cations such as antacids, calcium-rich foods, and mineral supplements
  • Take PROMACTA without a meal or with a meal low in calcium (≤50 mg)

Adverse Reactions
Across all indications, the most common adverse reactions (≥20% in any indication) were anemia, nausea, pyrexia, ALT increased, cough, fatigue, headache, and diarrhea.

The most common adverse reactions in 3 placebo-controlled clinical trials in patients with persistent or chronic ITP (≥3% and greater than placebo) for PROMACTA were nausea (9%), diarrhea (9%), upper respiratory tract infection (7%), vomiting (6%), increased ALT (5%), myalgia (5%), urinary tract infection (5%), oropharyngeal pain (4%), increased AST (4%), pharyngitis (4%), back pain (3%), influenza (3%), paresthesia (3%), and rash (3%).

The most common adverse reactions in 2 placebo-controlled clinical trials in patients with persistent or chronic ITP 1 year and older (≥3% and greater than placebo) for PROMACTA were upper respiratory tract infection (17%), nasopharyngitis (12%), cough (9%), diarrhea (9%), pyrexia (9%), abdominal pain (8%), oropharyngeal pain (8%), toothache (6%), ALT increased (6%), rash (5%), AST increased (4%), and rhinorrhea (4%).

Please see full Prescribing Information for PROMACTA, including Boxed WARNING, and Medication Guide.