Immune Thrombocytopenia

POLICY NUMBER

L.5.01.609

Doptelet (avatrombopag)Gammagard (intravenous IgG)Gammagard S/D (intravenous IgA)Gammaked (intravenous IgG)Gammaplex (intravenous IgG)Gamunex-C (intravenous IgG)Octagam (intravenous IgG)Privigen (intravenous IgG)Promacta (eltrombopag)Riabni (rituximab-arrx)Rituxan (rituximab)Ruxience (rituximab-pvvr)Tavalisse (fostamatinib disodium hexahydrate)Truxima (rituximab-abbs)Please perform a formulary drug search on your patient’s member ID to ensure the prescription drug is covered under their benefit plan. The medication(s) in this medical policy may not be covered under a specific member’s benefit plan.

DESCRIPTION

Immune thrombocytopenia (ITP, also called idiopathic thrombocytopenic purpura) is an acquired thrombocytopenia characterized by immune-mediated destruction of platelets and/or impairment of platelet production. The condition may happen in isolation (primary) or in association with other disorders (secondary). Some secondary causes are autoimmune diseases (e.g., rheumatoid arthritis, systemic lupus erythematosus), immunodeficiency syndromes (e.g., HIV), infections (e.g., cytomegalovirus [CMV], Epstein-Barr virus [EBV], hepatitis C), and medication induced (e.g., alemtuzumab, ipilimumab, valproic acid). ITP is categorized into the following three phases:

• newly-diagnosed (within 3 months of diagnosis)

• persistent (between 3 and 12 months of diagnosis)

• chronic (condition present for >12 months). 

Related medical policies:

• Biosimilar Medications

• Immunoglobulin Therapy

• Promacta (eltrombopag)

• Rituximab Non-Oncology

POLICY

Prior authorization is required.

The use of samples by an individual will not be considered current or stable therapy to satisfy Medical Policy requirements.

Initial Criteria

Treatment of immune thrombocytopenia (ITP) may be considered medically necessary when ALL of the following criteria are met:

1. The request is for a formulary rituximab biosimilar and BOTH of the following:

   1. The individual has a documented diagnosis of acute or chronic immune thrombocytopenia (ITP);

   2. The individual’s platelet count is <30,000µL; AND

   3. ONE of the following:

      1. The individual had an inadequate response to corticosteroids AND immunoglobulin therapy (i.e., failure to achieve and maintain a platelet count ≥30,000/µL for 3-6 months following completion of treatment) with or without a splenectomy; OR

      2. The individual has a documented intolerance, FDA labeled contraindication, or hypersensitivity to one corticosteroid AND immunoglobulin therapy;

2. The request is for intravenous IgG and ALL of the following:

   1. The request is for ONE of the following:

      1. Gammaked or Gamunex-C;

      2. Privigen, and the individual is ≥15 years of age; OR

      3. Octagam, and the individual is ≥18 years of age;

   2. ONE of the following:

      1. The individual has a documented diagnosis of acute immune thrombocytopenia (see Policy Description) with ONE of the following:

         1. major bleeding (e.g., life-threatening bleeding and/or clinically important mucocutaneous bleeding);

         2. severe thrombocytopenia (platelet <20,000 per microliter [adult] or 30,000 per microliter [child]) and at high risk for bleeding complications;

         3. severe thrombocytopenia (platelet <20,000 per microliter [adult] or 30,000 per microliter [child]) and a slow or inadequate response to corticosteroids; OR

         4. severe thrombocytopenia (platelet <20,000 per microliter [adult] or 30,000 per microliter [child]) and a predictable risk of bleeding in the future (e.g., a procedure or surgery with a high bleeding risk);

      2. The individual has a diagnosis of persistent to chronic ITP (see Policy Description) and ALL of the following:

         1. at least 6 months of disease duration;

         2. persistent thrombocytopenia (i.e., platelets <20,000/µL or <30,000/µL with severe bleeding [e.g., intracranial, gastrointestinal]); AND

         3. inadequate response to corticosteroids (i.e., failure to achieve and maintain a platelet count ≥30,000/µL for 3-6 months following completion of treatment); AND

   3. ONE of the following:

      1. The individual has had an inadequate response to corticosteroids (i.e., failure to achieve and maintain a platelet count ≥30,000/µL for 3-6 months following completion of treatment); OR

      2. The individual has had an inadequate response to splenectomy;

3. The request is for Promacta (eltrombopag) and ALL of the following:

   1. The individual is ≥1 year of age;

   2. The individual has a diagnosis of persistent or chronic immune thrombocytopenia (ITP);

   3. ONE of the following:

      1. The individual has a baseline platelet count <30,000/µL (30 x10⁹/L) with severe bleeding (e.g., intracranial, gastrointestinal); OR

      2. The individual has a baseline platelet count of <20,000/µL (20 x10⁹/L); AND

   4. ONE of the following:

      1. The individual has had an inadequate response (i.e., failure to achieve and maintain a platelet count ≥30,000/µL (30 x10⁹/L) for 3-6 months following completion of treatment) to all of the following therapies, with or without a splenectomy:

         1. corticosteroids;

         2. immunoglobulins (IVIG);

         3. Rituximab;

      2. The individual has a documented intolerance, FDA-labeled contraindication, or hypersensitivity to ALL of the above treatments;

      3. The individual will not be prescribed combination therapy with thrombopoietin;

4. The request is for Tavalisse (fostamatinib disodium hexahydrate) and ALL of the following:

   1. The individual is 18 years of age or older;

   2. The individual has a documented diagnosis of chronic (>12 months since diagnosis) immune thrombocytopenia (ITP);

   3. ONE of the following:

      1. The individual has a baseline platelet count <30,000/µL with severe bleeding (e.g., intracranial, gastrointestinal); OR

      2. The individual has a baseline platelet count <20,000/µL;

   4. The individual has had an inadequate response (i.e., failure to achieve and maintain a platelet count ≥30,000/µL for 3-6 months following completion of treatment) to ALL of the following therapies, with or without a splenectomy:

      1. Corticosteroids;

      2. Immunoglobulin therapy;

      3. Rituximab; AND

      4. Promacta (eltrombopag);

   5. The prescriber will monitor blood pressure, liver enzymes (including ALT, AST and bilirubin), and CBC monthly; AND

   6. The individual will not be prescribed combination therapy with thrombopoietin receptor agonists (i.e., Promacta, Nplate, Doptelet, Mulpleta, etc.);

5. The prescriber is a specialist or has consulted with a specialist in an area of the individual’s diagnosis (i.e., a hematologist);

6. The individual does not have any FDA-labeled contraindication(s) to therapy with the requested agent; AND

7. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage. 

Length of Approval:

• Formulary IVIG: 12 months

• Formulary rituximab biosimilar: 12 months

• Promacta: 8 weeks

• Tavalisse: 3 months

Renewal Criteria

Treatment of immune thrombocytopenia (ITP) may be approved for RENEWAL when ALL of the following criteria are met:

1. The individual has been previously approved for therapy with the requested agent through BCBSMS PA process;

2. ONE of the following:

   1. The request is for formulary IVIG;

   2. The request is for formulary rituximab biosimilar;

   3. The request is for Promacta and ALL of the following:

      1. The individual has shown clinical improvement (i.e. increase in platelet count from baseline and at least doubling of the baseline platelet count);

      2. Current (within the last 90 days) platelet count is <400 x 10⁹/L; AND

      3. The individual is not being treated using combination therapy with thrombopoietin receptor agonists (e.g., Nplate, Doptelet, Mulpleta);

   4. The request is for Tavalisse and ALL of the following:

      1. The individual has had improvement in platelet count to ≥50,000/µL;

      2. The prescriber will monitor CBC, liver enzymes, and blood pressure monthly throughout therapy; AND

      3. The individual is not being treated using combination therapy with thrombopoietin receptor agonists (e.g., Promacta, Nplate, Doptelet, Mulpleta);

3. The prescriber is a specialist or has consulted with a specialist in an area of the individual’s diagnosis (i.e., hematologist);

4. The individual does not have any FDA-labeled contraindication(s) to therapy with the requested agent; AND

5. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage. 

Length of Approval:

• Formulary IVIG: 12 months

• Formulary rituximab biosimilar: 12 months

• Promacta: 12 months

• Tavalisse: 12 months

The following medications are considered not medically necessary for the treatment of Immune Thrombocytopenia (ITP) as there are other alternatives covered by the Plan for the treatment of this condition:

• Doptelet (avatrombopag)

• Gammagard S/D (intravenous IgA)

• Gammaplex (Immune Globulin Intravenous (Human) 5% Liquid)

• Riabni (rituximab-arrx)

• Rituxan (rituximab)

Services related to delivery and/or administration of a medication which have not been approved through the BCBSMS PA review process will be considered not medically necessary.

POLICY EXCEPTIONS

State Health Plan (State and School Employees) Participants

POLICY GUIDELINES

The coverage guidelines outlined in the Medical Policy Manual should not be used in lieu of the Member's specific benefit plan language.

Medically Necessary is defined as those services, treatments, procedures, equipment, drugs, devices, items or supplies furnished by a covered Provider that are required to identify or treat a Member's illness, injury or Mental Health Disorders, and which Company determines are covered under this Benefit Plan based on the criteria as follows in A through D:

1. consistent with the symptoms or diagnosis and treatment of the Member's condition, illness, or injury; and

2. appropriate with regard to standards of good medical practice; and

3. not solely for the convenience of the Member, his or her Provider; and

4. the most appropriate supply or level of care which can safely be provided to Member. When applied to the care of an Inpatient, it further means that services for the Member's medical symptoms or conditions require that the services cannot be safely provided to the Member as an Outpatient.

For the definition of medical necessity, “standards of good medical practice” means standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, and physician specialty society recommendations, and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors. BCBSMS makes no payment for services, treatments, procedures, equipment, drugs, devices, items or supplies which are not documented to be Medically Necessary. The fact that a Physician or other Provider has prescribed, ordered, recommended, or approved a service or supply does not in itself, make it Medically Necessary.

BCBSMS may request medical records for determination of medical necessity. When medical records are requested, letters of support and/or explanation are often useful, but are not sufficient documentation unless all specific information needed to make a medical necessity determination is included.

Medication Failure

Medication failure is defined as disease progression at generally accepted doses at generally accepted durations of therapy, as outlined in medical policy, as appropriate for the disease state being treated. Dosages below the recommended dose and duration for the specific condition being treated and/or experience of common side effects of medication will not be considered medication failure or lack of response for the purpose of this review.

BCBSMS determines patient medication trial and adherence by a review of pharmacy claims data over the preceding twelve months. Additional information may be requested on a case-by-case basis to allow for proper review. If individual is new to BCBSMS and pharmacy records are needed to confirm medication trials and adherence, it is the responsibility of the individual and/or requesting provider to obtain said records and to submit them to BCBSMS upon request. Medical records from the provider that list previously prescribed medications will not be sufficient to show medication trials or adherence.

POLICY HISTORY

11/01/2024: New policy added. Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee. Effective 01/01/2025.

01/01/2025: Code Reference section updated to add new HCPCS code J2802.

02/18/2025: Policy updated to remove Nplate (romiplostim) from the list of not medically necessary medications. Sources updated. Code Reference section updated to remove HCPCS code J2796 and J2802 from the Not Medically Necessary Codes.

03/21/2025: Policy section updated to remove Truxima (rituximab-abbs) from the list of not medically necessary medications. Code Reference section updated to move HCPCS code Q5115 from the Not Medically Necessary Codes table to the Medically Necessary Codes table.

09/09/2025: Policy reviewed by Pharmacy & Therapeutics (P&T) Committee; no changes to policy statement. Updated the definition of Medication Failure in Policy Guidelines. Sources updated.

SOURCE(S)

1. Algazy KM. Idiopathic Thrombocytopenic Purpura. In: NORD Guide to Rare Disorders. Lippincott Williams & Wilkins. Philadelphia, PA. 2003:415-16.

2. Arnold, D., Cuker, A.

   Immune

   thrombocytopenia

   (ITP) in adults: Clinical manifestations and diagnosis. In: UpToDate, Connor RF (Ed), Wolters Kluwer. Accessed on June 24, 2025. Available from

   https://www.uptodate.com/contents/immune-thrombocytopenia-itp-in-adults-clinical-manifestations-and-diagnosis

3. Arnold, D., Cuker, A.

   Initial treatment of immune thrombocytopenia (ITP) in adults

   . In: UpToDate, Connor RF (Ed), Wolters Kluwer. Accessed on June 24, 2025. Available from

   https://www.uptodate.com/contents/initial-treatment-of-immune-thrombocytopenia-itp-in-adults

4. Blue Cross & Blue Shield Association Policy # 5.01.24

   Off Label Use of Rituximab

5. Blue Cross Blue Shield Association Policy # 8.01.05

   Immunoglobulin Therapy

6. Doptelet prescribing information. AkaRx, Inc.

   July 2024. Last accessed June 2025.

7. Gammagard S/D prescribing information. Takeda Pharmaceuticals America, Inc.

   March 2025. Last accessed June 2025.

8. Gammaked prescribing information. Kedrion Biopharma, Inc.

   January 2020. Last accessed June 2025.

9. Gammaplex prescribing information. Bio Products Laboratory Limited.

   October 2024. Last accessed June 2025.

10. Gamunex-C prescribing information. Grifols USA, LLC.

    April 2022. Last accessed June 2025.

11. Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for

    immune

    thrombocytopenia

    .

    Blood Adv

    2019; 3:3829-3866. doi: 

    https://doi.org/10.1182/bloodadvances.2019000966

12. Octagam prescribing information. Pfizer Laboratories Div Pfizer Inc.

    January 2024. Last accessed June 2025.

13. Privigen prescribing information. CSL Behring AG.

    May 2025. Last accessed June 2025.

14. Promacta prescribing information. Novartis Pharmaceuticals Corporation.

    June 2025. Last accessed June 2025.

15. Riabni prescribing information. Amgen, Inc.

    February 2024. Last accessed June 2025.

16. Rituxan prescribing information. Genentech, Inc.

    January 2025. Last accessed June 2025.

17. Ruxience prescribing information. Pfizer Laboratories Div. Pfizer, Inc.

    June 2025. Last accessed June 2025.

18. Tavalisse prescribing Information. Rigel Pharmaceuticals, Inc.

    August 2024. Last accessed June 2025.

19. Truxima prescribing information. Cephalon, Inc.

    June 2025. Last accessed June 2025.

20. Zaja F, Vianelli N, Volpetti S, Battista ML, Defina M, Palmieri S, Bocchia M, Medeot M, De Luca S, Ferrara F, Isola M, Baccarani M, Fanin R. Low-dose rituximab in adult patients with primary immune thrombocytopenia. Eur J Haematol. 2010 Oct;85(4):329-34. doi: 10.1111/j.1600-0609.2010.01486.x. Epub 2010 Jul 28. PMID: 20546023.

CODE REFERENCE

This may not be a comprehensive list of procedure codes applicable to this policy.

The code(s) listed below are ONLY medically necessary if the procedure is performed according to the "Policy" section of this document.

Medically Necessary Codes

Not Medically Necessary Codes

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