Last Review Date: 02/01/2024

Date of Origin: 01/01/2012

Dates Reviewed: 12/2011, 02/2013, 02/2014, 12/2014, 10/2015, 09/2016, 12/2016, 03/2017, 06/2017, 12/2017, 03/2018, 06/2018, 10/2018, 01/2019, 12/2019, 02/2020, 02/2021, 02/2022, 02/2023, 02/2024

1. Length of Authorization ¹

Coverage will be provided for 3 months and may be renewed, unless otherwise specified.

• Coverage for use to treat Hematopoietic Syndrome of Acute Radiation Syndrome (HS­ARS) cannot be renewed.

2. Dosing Limits

A. Quantity Limit (max daily dose) [NDC Unit]:

• Nplate 125 mcg single-dose vial for injection: 40 vials per 28 days
• Nplate 250 mcg single-dose vial for injection: 20 vials per 28 days
• Nplate 500 mcg single-dose vial for injection: 12 vials per 28 days

B. Max Units (per dose and over time) [HCPCS Unit]:

• ITP and CIT: 125 billable units weekly
• MDS: 100 billable units weekly
• HS­ARS: 125 billable units x 1 dose

3. Initial Approval Criteria ¹

Coverage is provided in the following conditions:

Universal Criteria ¹

• Patient is not on any other thrombopoietin receptor agonist or mimetic (e.g., lusutrombopag, eltrombopag, avatrombopag, etc.) or fostamatinib; AND
• Romiplostim is not being used to attempt to normalize platelet count (i.e., use is limited to decreasing the risk of bleeding from thrombocytopenia by increasing platelet levels and not normalizing them); AND
• Laboratory values for platelet count are current (i.e., drawn within the previous 28 days);** AND

**NOTE: Does not apply to patients receiving treatment for Hematopoietic Syndrome of Acute Radiation Syndrome (HS-ARS)

Immune (idiopathic) Thrombocytopenia (ITP) † Ф ^1,5

• The patient is at increased risk for bleeding as indicated by platelet count less than 30 × 10⁹/L (30,000/mm³); AND
  • Patient has acute ITP; AND
    • Patient is at least 18 years of age; AND
    • Patient has previously failed any of the following treatments for ITP:
      • Patient has failed previous therapy with corticosteroids; OR
      • Patient has failed previous therapy with immunoglobulins; OR
      • Patient has had a splenectomy; OR
  • Patient has had chronic ITP for at least 6 months (or meets the corticosteroid requirement below); AND
    • Patient is at least 1 year of age; AND
    • Patient has previously failed any of the following treatments for ITP:
      • Patient has failed previous therapy with corticosteroids (i.e., patient had no response to at least a 3-month trial or is corticosteroid-dependent); OR
      • Patient has failed previous therapy with immunoglobulins; OR
      • Patient has had a splenectomy

Hematopoietic Syndrome of Acute Radiation Syndrome (HS-ARS) † Ф ¹

• Patient has suspected or confirmed exposure to radiation levels greater than 2 gray (Gy)

Chemotherapy-Induced Thrombocytopenia (CIT) ‡ ^2,15-19

• Patient is at least 18 years of age; AND
• Patient has a platelet count less than 100 × 10⁹/L (100,000/mm³) for at least 3 to 4 weeks after the last chemotherapy administration and/or after delays in chemotherapy initiation related to thrombocytopenia

Myelodysplastic Syndromes (MDS) ‡ ^{2,3,13,14,20,21}

• Patient is at least 18 years of age; AND
• Patient has lower risk disease [i.e., IPSS-R (Very Low, Low, Intermediate)]; AND
• Patient has severe or refractory thrombocytopenia (i.e., platelet count <50 x 10⁹/L); AND
• Patient progressed or had no response to hypomethylating agents (e.g., azacitidine, decitabine, etc.) or immunosuppressive therapy

† FDA Approved Indication(s); ‡ Compendia Recommended Indication(s); Ф Orphan Drug

4. Renewal Criteria ¹

Coverage can be renewed based upon the following criteria:

• Patient continues to meet the universal and other indication-specific relevant criteria such as concomitant therapy requirements (not including prerequisite therapy), performance status, etc. identified in section III; AND
• Absence of unacceptable toxicity from the drug. Examples of unacceptable toxicity include: thrombotic/thromboembolic complications, risk of progression of myelodysplastic syndromes to acute myelogenous leukemia, loss of response to romiplostim/presence of neutralizing antibodies to romiplostim, etc.; AND

Immune (idiopathic) Thrombocytopenia (ITP) † ¹

• Disease response as indicated by the achievement and maintenance of a platelet count of at least 50 × 10⁹/L (not to exceed 400 x 10⁹/L) as necessary to reduce the risk for bleeding

Hematopoietic Syndrome of Acute Radiation Syndrome (HS-ARS) † ¹

• Coverage cannot be renewed

Chemotherapy-Induced Thrombocytopenia (CIT) ‡ ^2,15-19

• Patient continues to receive chemotherapy; AND
• Disease response as indicated by the achievement and maintenance of a platelet count of at least 100 × 10⁹/L (not to exceed 400 x 10⁹/L)

Myelodysplastic Syndromes (MDS) ‡ ^2,3,21

• Patient has not developed acute myeloid leukemia (AML) (Note: romiplostim induces an increase in immature white blood cells and peripheral blasts which is not indicative of development of AML); AND

• Disease response as indicated by an increase in platelet count compared to pretreatment baseline (not to exceed 450 x 10⁹/L), reduction in bleeding events, or reduction in platelet transfusion requirements

5. Dosage/Administration ^1,3,19,21

6. Billing Code/Availability Information

HCPCS Code:

• J2796 – Injection, romiplostim, 10 micrograms; 10 mcg = 1 billable unit

NDC(s):

• Nplate 125 mcg single-dose vial: 55513-0223-xx
• Nplate 250 mcg single-dose vial: 55513-0221-xx
• Nplate 500 mcg single-dose vial: 55513-0222-xx

7. References

1. Nplate [package insert]. Thousand Oaks, CA; Amgen Inc; February 2022. Accessed January 2024.
2. Referenced with permission from the NCCN Drugs & Biologics Compendium (NCCN Compendium®) for romiplostim. National Comprehensive Cancer Network, 2024. The NCCN Compendium® is a derivative work of the NCCN Guidelines®. NATIONAL COMPREHENSIVE CANCER NETWORK®, NCCN®, and NCCN GUIDELINES® are trademarks owned by the National Comprehensive Cancer Network, Inc. To view the most recent and complete version of the Compendium, go online to www.nccn.org/. Accessed January 2024.
3. Giagounidis A, Mufti GJ, Fenaux P, et al. Results of a randomized, double-blind study of romiplostim versus placebo in patients with low/intermediate-1-risk myelodysplastic syndrome and thrombocytopenia. Cancer. 2014 Jun 15;120(12):1838-46.
4. Lambert MP, Gernsheimer TB. Clinical updates in adult immune thrombocytopenia. Blood. 2017. 129:2829-2835. Doi:10.1182/blood-2017-03-754119.
5. Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 Dec 10;3(23):3829-3866.
6. Newland A, Godeau B, Priego V, et al. Remission and platelet responses with romiplostim in primary immune thrombocytopenia: final results from a phase 2 study. Br J Haematol. 2016 Jan;172(2):262-73. Doi: 10.1111/bjh.13827.
7. Bussel JB, Buchanan GR, Nugent DJ, et al, “A Randomized, Double-Blind Study of Romiplostim to Determine Its Safety and Efficacy in Children With Immune Thrombocytopenia,” Blood, 2011, 118(1):28-36.
8. Bussel JB, Kuter DJ, Pullarkat V, et al, “Safety and Efficacy of Long-Term Treatment With Romiplostim in Thrombocytopenic Patients With Chronic ITP,” Blood, 2009, 113(10):2161-71.
9. Kuter DJ, Bussel JB, Lyons RM, et al, “Efficacy of Romiplostim in Patients With Chronic Immune Thrombocytopenic Purpura: A Double-Blind Randomized Controlled Trial,” Lancet, 2008, 371(9610):395-403.
10. Kuter DJ, Rummel M, Boccia R, et al, “Romiplostim or Standard of Care in Patients With Immune Thrombocytopenia,” N Engl J Med, 2010, 363(20):1889-99.
11. Kuter DJ, Bussel JB, Newland A, et al. Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy. Br J Haematol. 2013 May;161(3):411-23. Doi: 10.1111/bjh.12260.
12. Tarantino MD, Bussel JB, Blanchette VS, et al. Romiplostim in children with immune thrombocytopenia: a phase 3, randomized, double-blind, placebo-controlled study. Lancet. 2016 Jul 2;388(10039):45-54. Doi: 10.1016/S0140-6736(16)00279-8.
13. Kantarjian H, Fenaux P, Sekeres MA, et al. Safety and efficacy of romiplostim in patients with lower-risk myelodysplastic syndrome and thrombocytopenia. J Clin Oncol. 2010 Jan 20;28(3):437-44.
14. Kantarjian HM, Giles FJ, Greenberg PL, et al. Phase 2 study of romiplostim in patients with low- or intermediate-risk myelodysplastic syndrome receiving azacitidine therapy. Blood. 2010 Oct 28;116(17):3163-70.
15. Soff GA, Miao Y, Bendheim G, et al. Romiplostim Treatment of Chemotherapy-Induced Thrombocytopenia. J Clin Oncol. 2019 Nov 1;37(31):2892-2898. Doi: 10.1200/JCO.18.01931.
16. Al-Samkari H, Parnes AD, Goodarzi K, et al. A multicenter study of romiplostim for chemotherapy-induced thrombocytopenia in solid tumors and hematologic malignancies. Haematologica. 2021 Apr 1;106(4):1148-1157. Doi: 10.3324/haematol.2020.251900.
17. Miao J, Leblebjian H, Scullion B, et al. A single center experience with romiplostim for the management of chemotherapy-induced thrombocytopenia. Am J Hematol. 2018 Aug;93(4):E86-E88. Doi: 10.1002/ajh.25022.
18. Parameswaran R, Lunning M, Mantha S, et al. Romiplostim for management of chemotherapy-induced thrombocytopenia. Support Care Cancer. 2014 May;22(5):1217-22. Doi: 10.1007/s00520-013-2074-2.
19. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines^®) for Hematopoietic Growth Factors 2.2024. National Comprehensive Cancer Network, 2024. NATIONAL COMPREHENSIVE CANCER NETWORK®, NCCN®, and NCCN GUIDELINES® are trademarks owned by the National Comprehensive Cancer Network, Inc.” To view the most recent and complete version of the Guidelines, go online to NCCN.org. Accessed January 2024.
20. Sekeres MA, Kantarjian H, Fenaux P, et al. Subcutaneous or intravenous administration of romiplostim in thrombocytopenic patients with lower risk myelodysplastic syndromes. Cancer. 2011 Mar 1;117(5):992-1000. doi: 10.1002/cncr.25545. Epub 2010 Oct 13. PMID: 20945323.
21. Fenaux P, Muus P, Kantarjian H, et al. Romiplostim monotherapy in thrombocytopenic patients with myelodysplastic syndromes: long-term safety and efficacy. Br J Haematol. 2017 Sep;178(6):906-913. doi: 10.1111/bjh.14792. Epub 2017 Jun 14. PMID: 28616874; PMCID: PMC5600084.
22. CGS Administrators, LLC. Local Coverage Article: Billing and Coding: Immune Thrombocytopenia (ITP) Therapy (A57160). Centers for Medicare & Medicaid Services, Inc. Updated on 11/07/2023 with effective date 11/16/2023. Accessed January 2024.

Appendix 1 – Covered Diagnosis Codes

Appendix 2 – Centers for Medicare and Medicaid Services (CMS)

The preceding information is intended for non-Medicare coverage determinations. Medicare coverage for outpatient (Part B) drugs is outlined in the Medicare Benefit Policy Manual (Pub. 100-2), Chapter 15, §50 Drugs and Biologicals. In addition, National Coverage Determinations (NCDs) and/or Local Coverage Determinations (LCDs) may exist and compliance with these policies is required where applicable. Local Coverage Articles (LCAs) may also exist for claims payment purposes or to clarify benefit eligibility under Part B for drugs which may be self-administered. The following link may be used to search for NCD, LCD, or LCA documents: https://www.cms.gov/medicare-coverage-database/search.aspx. Additional indications, including any preceding information, may be applied at the discretion of the health plan.

Medicare Part B Covered Diagnosis Codes (applicable to existing NCD/LCD/LCA):