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Table 68: Thrombocytopenic Agents


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Drug Category: Blood and Circulation

Medication Class/Individual Agents: Thrombocytopenic Agents

I. Prior-Authorization Requirements

 Thrombocytopenic Agents – Monoclonal Antibody

Clinical Notes

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes

caplacizumab-yhdp Cablivi PA  

Please note: In the case where the prior authorization (PA) status column indicates PA, both the brand and generic (if available) require PA. Typically, the generic is preferred when available unless the brand-name drug appears on the MassHealth Brand Name Preferred Over Generic Drug List. In general, when requesting the non-preferred version, whether the brand or generic, the prescriber must provide medical records documenting an inadequate response or adverse reaction to the preferred version, in addition to satisfying the criteria for the drug itself.

  • Thrombopoietin agonists are approved for the treatment of refractory thrombocytopenia in those patients who have had an insufficient response to corticosteroids, immunoglobulin, or splenectomy.
  • Eltrombopag is also approved for the treatment of severe aplastic anemia and thrombocytopenia in the setting of hepatitis C.
  • Romiplostim is also approved for the treatment of hematopoietic syndrome of acute radiation syndrome.
  • These agents are not approved for the normalization of platelet counts and should only be used in those whose clinical condition is associated with a high risk of bleeding.
  • Fostamatinib is a tyrosine kinase inhibitor with demonstrated activity against spleen tyrosine kinase (SYK). It is approved for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.
  • Avatrombopag and lusutrombopag are indicated for the treatment of thrombocytopenia in adults with chronic liver disease (CLD) who are scheduled to undergo a procedure.
    • For avatrombopag, dosing should begin 10-to-13 days before scheduled procedure. Patients should undergo their procedure five-to-eight days after the last dose.
    • For lusutrombopag, dosing should begin eight-to-14 days before scheduled procedure. Patients should undergo their procedure two-to-eight days after the last dose.
  • Avatrombopag is also indicated for thrombocytopenia in adults with chronic ITP who have had insufficient response to a previous treatment.
  • Caplacizumab-yhdp is a novel humanized immunoglobulin (nanobody) that works by targeting platelet (PLT) aggregation through binding to von Willebrand factor (vWF) and inhibiting interaction between vWF and PLTs. It is approved for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange (PEX) and immunosuppressive therapy. This agent should be administered upon initiation of PEX and continued once daily for 30 days following the last daily PEX.
 

 Thrombocytopenic Agents – Thrombopoietin Agonists

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes

avatrombopag Doptelet PA  
eltrombopag Promacta PA  
lusutrombopag Mulpleta PA  
romiplostim Nplate PA   MB

 Thrombocytopenic Agents – Tyrosine Kinase Inhibitor

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes

fostamatinib Tavalisse PA  
Table Footnotes
MB This drug is available through the health care professional who administers the drug or in an outpatient or inpatient hospital setting. MassHealth does not pay for this drug to be dispensed through the retail pharmacy. If listed, PA does not apply through the hospital outpatient and inpatient settings. Please refer to 130 CMR 433.408 for PA requirements for other health care professionals. Notwithstanding the above, this drug may be an exception to the unified pharmacy policy; please refer to respective MassHealth Accountable Care Partnership Plans (ACPPs) and Managed Care Organizations (MCOs) for PA status and criteria, if applicable.
 

II. Therapeutic Uses

FDA-approved, for example:

  • aTTP (Cablivi)
  • Chronic, relapsed, or refractory ITP (Doptelet, Nplate, Promacta, Tavalisse)
  • Hematopoietic syndrome of acute radiation ayndrome (HS-ARS)/acute exposure to myelosuppressive doses of radiation (Nplate)
  • Severe aplastic anemia (Promacta)
  • Thrombocytopenia due to CLD (Doptelet, Mulpleta)
  • Thrombocytopenia in the setting of hepatitis C with interferon therapy (Promacta)

Non-FDA-approved, for example:

  • Thrombocytopenia in the setting of hepatitis C independent of interferon therapy (Promacta)

Note: The above lists may not include all FDA-approved and non-FDA approved indications.

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III.  Evaluation Criteria for Approval

Please note: In the case where the prior authorization (PA) status column indicates PA, both the brand and generic (if available) require PA. Typically, the generic is preferred when available unless the brand-name drug appears on the MassHealth Brand Name Preferred Over Generic Drug List. In general, when requesting the non-preferred version, whether the brand or generic, the prescriber must provide medical records documenting an inadequate response or adverse reaction to the preferred version, in addition to satisfying the criteria for the drug itself.

  • All PA requests must include clinical diagnosis, drug name, dose, and frequency.
  • Dispensing in a 90-day supply of medication may be mandated or allowable for agents in this therapeutic class (designated by M90 or A90, respectively, in the Drug Notes section above). Applicable quantity limits are described below as units per day, per month, per 28 days, or as clinically appropriate, and may be extrapolated for fills of longer day supply. 
  • A preferred drug may be designated for this therapeutic class. In general, MassHealth requires a trial of the preferred drug or clinical rationale for prescribing a non-preferred drug within a therapeutic class. Additional information about these agents, including PA requirements and preferred products, can be found within the MassHealth Drug List at www.mass.gov/druglist.
  • For recertification requests, approval may require submission of additional documentation including, but not limited to, documentation of: some or all criteria for the original approval; response to therapy; clinical rationale for continuation of use; status of member’s condition; appropriate diagnosis; appropriate age; appropriate dose, frequency, and duration of use for requested medication; complete treatment plan; current laboratory values; and member’s current weight.
  • Additional criteria may apply depending upon diagnosis and/or requested medication (see below). Other factors, including rebate and FDA-approval status, may change independently of scheduled MassHealth updates, which may result in additional restrictions.

 

Cablivi

  • Documentation of the following is required for a diagnosis of aTTP:
    • appropriate diagnosis; and
    • member is ≥ 18 years of age; and
    • requested agent will be used initially in conjunction with immunosuppressive therapy; and
    • requested quantity is ≤ one unit/day after initial bolus injection.

  

Doptelet

  • Documentation of the following is required for a diagnosis of chronic, relapsed, or refractory ITP:
    • appropriate diagnosis; and
    • member is ≥ 18 years of age; and
    • one of the following:
      • platelet count < 30,000 cells/mcL; or
      • medical necessity for platelet elevation (upcoming surgery, peptic ulcer disease or condition that may predispose member to bleeding); and
    • inadequate response, adverse reaction, or contraindication to Promacta; and
    • requested quantity is ≤ two units/day; and
    • one of the following:
      • inadequate response or adverse reaction to one or contraindication to both of the following: corticosteroid, immunoglobulin; or
      • member has had a splenectomy.

 

  • Documentation of the following is required for a diagnosis of thrombocytopenia due to CLD:
    • appropriate diagnosis; and
    • member is ≥ 18 years of age; and
    • member is scheduled to undergo a procedure and date is provided; and
    • platelet count < 50,000 cells/mcL; and
    • one of the following:
      • if platelet count is 40,000 to < 50,000 cells/mcL, requested dose is 40 mg (two tablets) once daily for five days; or
      • if platelet count is less than 40,000 cells/mcL, requested dose is 60 mg (three tablets) once daily for five days.

   

Mulpleta

  • Documentation of the following is required for a diagnosis of thrombocytopenia due to CLD:
    • appropriate diagnosis; and
    • member is ≥ 18 years of age; and
    • member is scheduled to undergo a procedure and date is provided; and
    • platelet count < 50,000 cells/mcL; and
    • inadequate response, adverse reaction, or contraindication to Doptelet; and
    • requested dose is 3 mg once daily for a maximum of seven days.

   

Nplate

  • Documentation of the following is required for a diagnosis of chronic, relapsed, or refractory ITP:
    • appropriate diagnosis; and
    • member is ≥ one year of age; and
    • one of the following:
      • platelet count < 30,000 cells/mcL; or
      • medical necessity for platelet elevation (upcoming surgery, peptic ulcer disease or condition that may predispose member to bleeding); and
    • one of the following:
      • inadequate response or adverse reaction to one or contraindication to both of the following: corticosteroid, immunoglobulin; or 
      • member has had a splenectomy; and
    • inadequate response, adverse reaction, or contraindication to Promacta.

 

  • Documentation of the following is required for a diagnosis of HS-ARS/acute exposure to myelosuppressive doses of radiation:
    • appropriate diagnosis; and
    • requested dose is 10 mcg/kg for a one-time administration.

  

Promacta

  • Documentation of the following is required for a diagnosis of chronic, relapsed, or refractory ITP:
    • appropriate diagnosis; and
    • member is ≥ one year of age; and
    • one of the following:
      • platelet count < 30,000 cells/mcL; or
      • medical necessity for platelet elevation (upcoming surgery, peptic ulcer disease or condition that may predispose member to bleeding); and
    • requested dose is ≤ 75 mg/day; and 
    • for Promacta 12.5 mg, requested quantity is ≤ one unit/day; and
    • one of the following:
      • inadequate response or adverse reaction to one or contraindication to both of the following: corticosteroid, immunoglobulin; or
      • member has had a splenectomy.

 

  • Documentation of the following is required for a diagnosis of severe aplastic anemia:
    • appropriate diagnosis; and
    • member is ≥ two years of age; and
    • platelet count < 50,000 cells/mcL; and
    • requested dose is ≤ 150 mg/day; and 
    • for Promacta 12.5 mg, requested quantity is ≤ one unit/day; and
    • one of the following:
      • inadequate response, adverse reaction, or contraindication to immunosuppressive therapy with anti-thymocyte globulin (ATG) and cyclosporine; or
      • member is treatment naïve and the requested agent will be used in combination with ATG and cyclosporine.

 

  • Documentation of the following is required for a diagnosis of thrombocytopenia in the setting of hepatitis C with interferon therapy:
    • appropriate diagnosis; and
    • member is ≥ 18 years of age; and
    • requested dose is ≤100 mg/day; and
    • for Promacta 12.5 mg, requested quantity is ≤ one unit/day; and
    • one of the following:
      • member intends to initiate therapy with interferon and current platelet count is ≤ 75,000 cells/mcL; or
      • both of the following:
        • member has already begun interferon therapy and platelet count supports continued use; and
        • member has met criteria for continued interferon therapy based on treatment futility protocols per most recent PA for hepatitis antiviral agents.

 

  • Documentation of the following is required for a diagnosis of thrombocytopenia in the setting of hepatitis C independent of interferon therapy:
    • appropriate diagnosis; and
    • member is ≥ 18 years of age; and
    • requested dose is ≤100 mg/day; and
    • for Promacta 12.5 mg, requested quantity is ≤ one unit/day; and
    • current platelet count is ≤ 75,000 cells/mcL; and
    • member is not currently using interferon therapy and does not intend to begin therapy; and
    • inadequate response, adverse reaction, or contraindication to immunoglobulin.

 

Tavalisse

  • Documentation of the following is required for a diagnosis of chronic, relapsed, or refractory ITP:
    • appropriate diagnosis; and
    • member is ≥ 18 years of age; and
    • one of the following:
      • platelet count < 30,000 cells/mcL; or
      • medical necessity for platelet elevation (upcoming surgery, peptic ulcer disease or condition that may predispose member to bleeding); and
    • inadequate response, adverse reaction, or contraindication to Promacta; and
    • requested quantity is ≤ two units/day; and
    • one of the following:
      • inadequate response or adverse reaction to one or contraindication to both of the following: corticosteroid, immunoglobulin; or
      • member has had a splenectomy.


Original Effective Date: 03/2013

Last Revised Date: 03/2024


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Last updated 04/01/24

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