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Table 45: Beta Thalassemia, Myelodysplastic Syndrome, and Sickle Cell Disease Agents

A    B    C    D    E    F    G    H    I    J    K    L    M    N    O    P    Q    R    S    T    U    V    W    X    Y    Z

Drug Category: Blood Disorder Agents

Medication Class/Individual Agents: Hematopoietic Agents

I. Prior-Authorization Requirements

 Beta Thalassemia Agents - Erythroid Maturation Agents

Clinical Notes
Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes
luspatercept-aamt Reblozyl PA   MB

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.

 

  • Luspatercept-aamt is a subcutaneously (SC) administered erythroid maturation agent. It is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members of the Transforming Growth Factor-Beta (TGF-beta) superfamily involved in the late stages of erythropoiesis (red blood cell production).
    • FDA-approved dosing: 1 to 1.25 mg/kg SC every three weeks
    • This agent should be administered by a medical professional.
    • Luspatercept-aamt should be discontinued if one does not experience a decrease in transfusion burden after nine weeks of treatment at the maximum dose level or if unacceptable toxicity occurs at any time.
  • L-glutamine is an oral agent indicated to reduce acute complications in children ≥ five years of age and adults with sickle cell disease (SCD).
  • Guidelines from the British Society for Haematology and the National Heart, Lung, and Blood Institute (NHLBI) recommend the use of hydroxyurea for adults with SCD who have experienced three or more moderate to severe pain crises in a 12-month period, pain or chronic anemia interfering with daily activities or with severe or recurrent episodes of acute chest syndrome (ACS). In addition, they give a strong recommendation for use in children nine-to-42 months of age and a moderate recommendation for children and adolescents > 42 months of age regardless of disease severity.
  • NHLBI recommends aiming for target ANC ≥ 2,000/uL. Maintain PLT count ≥ 80,000/uL. If neutropenia or thrombocytopenia occurs, hold hydroxyurea and monitor complete blood count with WBC differential weekly. When blood counts have recovered, reinstitute hydroxyurea at 5 mg/kg/day and if warranted, increase by 5 mg/kg/day increments every eight weeks until mild myelosuppression (ANC 2,000 to 4,000/uL to a maximum dose of 35 mg/kg/day).
  • NHLBI notes that a clinical response to hydroxyurea may take three-to-six months. A six-month trial on the maximum tolerated dose is required prior to considering discontinuation due to treatment failure.1
  • Crizanlizumab-tmca is the first humanized anti-P-selectin monoclonal antibody FDA-approved to reduce the frequency of vasoocclusive crises (VOCs) in adults and pediatric patients aged 16 years and older with sickle cell disease.
    • It is given as an intravenous (IV) infusion at a dose of 5 mg/kg at week 0, week 2 and then every four weeks.
    • This agent should be administered by a medical professional.
  • Voxelotor is an oral HbS polymerization inhibitor indicated for the treatment of SCD in adults and pediatric patients four years of age and older.
  • Betibeglogene autotemcel is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions This agent is a one-time intravenous (IV) infusion designed to deliver a functional copy of human β-globin gene (βA-T87Q-globin) into a patient’s own hematopoietic stem cells (HSC). Given the risk of serious adverse reactions, this agent is administered only by qualified treatment centers.
    • MassHealth Drug Utilization Review will be reaching out to prescribers after administration to verify clinical effectiveness and at ongoing intervals for long-term monitoring of sustained response. For additional information regarding betibeglogene autotemcel, please see the Acute Hospital Carve-Out Drugs List found at www.mass.gov/druglist.
  • Exagamglogene autotemcel is an autologous, ex vivo CRISPR/Cas9 gene-editing therapy indicated for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs). This agent is a one-time IV infusion that works to edit the erythroid-specific enhancer region of BCL11A in the CD34+ hematopoietic stem and progenitor cells (HSPCs) to reduce erythroid-specific expression of BCL11A and thereby increase levels of fetal hemoglobin (HbF). Given the risk of serious adverse reactions, this agent is administered only by qualified treatment centers.
    • MassHealth Drug Utilization Review will be reaching out to prescribers after administration to verify clinical effectiveness and at ongoing intervals for long-term monitoring of sustained response. For additional information regarding exagamglogene, please see the Acute Hospital Carve-Out Drugs List found at www.mass.gov/druglist.

1.Yawn BP, Buchanan GR, Afenyi-Annan AN, Ballas SK, Hassell KL, James AH, et al. Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members. JAMA. 2014 Sep 10;312(10):1033-48.

 

 Beta Thalassemia Agents - Gene Therapy
Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes
betibeglogene autotemcel Zynteglo PD PA   CO, MB

 Sickle Cell Disease Agents - Antimetabolites
Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes
hydroxyurea capsule Droxia test  
hydroxyurea tablet Siklos PA  

 Sickle Cell Disease Agents - Gene Therapy
Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes
exagamglogene autotemcel for sickle cell disease Casgevy PD PA   CO, MB

 Sickle Cell Disease Agents - Hemoglobin S (HbS) Polymerization Inhibitor
Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes
voxelotor Oxbryta PA  

 Sickle Cell Disease Agents - Not Otherwise Classified
Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes
l-glutamine Endari PA  

 Sickle Cell Disease Agents - P-Selectin Inhibitors
Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes
crizanlizumab-tmca Adakveo PA   MB
Table Footnotes
CO Carve-Out. This agent is listed on the Acute Hospital Carve-Out Drugs List and is subject to additional monitoring and billing requirements.
 
PD Preferred Drug. In general, MassHealth requires a trial of the preferred drug or clinical rationale for prescribing a non-preferred drug within a therapeutic class.
 
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:  

  • Beta thalassemia (Reblozyl, Zynteglo)
  • Myelodysplastic syndromes associated anemia (Reblozyl)
  • Sickle cell disease (Adakveo, Casgevy, Endari, Oxbryta, Siklos)

Note: The above list may not include all 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.

 

Adakveo

  • Documentation of all of the following is required: 
    • diagnosis of sickle cell disease; and
    • member is ≥ 16 years of age; and
    • prescriber is a hematologist or consult notes from a hematologist are provided; and
    • member has experienced two or more sickle cell crises in the previous 12 months; and
    • member's current weight; and
    • inadequate response to hydroxyurea at the maximally tolerated dose for at least three months; or
    • adverse reaction or contraindication to hydroxyurea.
  • For recertification, documentation of positive response to therapy (e.g., decrease in VOCs, reduction in need for pain management, decrease in hospitalizations) is required.

 

Casgevy

  • Documentation of all of the following is required: 
    • diagnosis of sickle cell disease; and
    • copy of genetic test confirming diagnosis of SCD; and
    • prescriber is a hematologist or consult notes from a hematologist are provided; and
    • member is ≥ 12 years of age; and
    • history of ≥ two sickle cell crises per year in the last two years; and
    • one of the following:
      • inadequate response to hydroxyurea therapy at the maximally tolerated dose for at least three months*; or
      • adverse reaction or contraindication to hydroxyurea; and 
    • appropriate dosing and treatment dates; and
    • infusion will take place in a qualified treatment facility; and
    • member is clinically stable and eligible for HSCT; and
    • member does not have active HIV, HBV, or HCV infection; and
    • member has not received any prior SCD gene therapy.

* Requests will be evaluated taking into account MassHealth pharmacy claims history or additional documentation addressing adherence to these agents.

 

Endari

  • Documentation of all of the following is required: 
    • diagnosis of sickle cell disease; and
    • member is ≥ five years of age; and
    • prescriber is a hematologist or consult notes from a hematologist are provided; and
    • member has experienced two or more sickle cell crises in the previous 12 months; and
    • member's current weight; and
    • inadequate response, adverse reaction, or contraindication to hydroxyurea.

 

Oxbryta

  • Documentation of all of the following is required: 
    • diagnosis of sickle cell disease; and
    • member is ≥ four years of age; and
    • prescriber is a hematologist or consult notes from a hematologist are provided; and
    • member has experienced at least one sickle cell crisis in the previous 12 months; and
    • one of the following:
      • inadequate response to hydroxyurea therapy at the maximally tolerated dose for at least three months; or
      • adverse reaction or contraindication to hydroxyurea; and
    • member has a baseline hemoglobin level ≤ 10.5 g/dL (lab work should be drawn within the last 60 days); and
    • for Oxbryta 300 mg tablet for oral suspension, medical necessity for the requested formulation as noted by one of the following:
      • member is < 13 years of age; or
      • member utilizes tube feeding (G-tube/J-tube); or
      • member has a swallowing disorder or condition affecting ability to swallow; and
    • appropriate dosing.
  • For recertification, documentation of positive response to therapy (e.g., decrease in VOCs, Hb increase of at least one g/dL from baseline, reduction in laboratory markers associated with hemolysis) is required.

   

Reblozyl for beta thalassemia

  • Documentation of all of the following is required: 
    • medical records and genetic testing supporting diagnosis of transfusion-dependent beta thalassemia; and
    • member is ≥ 18 years of age; and
    • prescriber is a hematologist or consult notes from a hematologist supporting the use of the requested agent are provided; and
    • member's current weight.
  • For recertification, documentation of positive response to therapy (e.g., decrease in transfusion requirements) is required.

 

Reblozyl for myelodysplastic syndromes associated anemia

  • Documentation of all of the following is required: 
    • appropriate diagnosis; and
    • member is ≥ 18 years of age; and
    • prescriber is a hematologist or consult notes from a hematologist supporting the use of the requested agent are provided; and
    • member's current weight.
  • For recertification, documentation of positive response to therapy (e.g., decrease in transfusion requirements) is required.

 

Siklos

  • Documentation of all of the following is required: 
    • diagnosis of sickle cell disease; and
    • member is ≥ two years of age; and
    • prescriber is a hematologist or consult notes from a hematologist are provided; and
    • member's current weight; and
    • medical necessity for the requested formulation as noted by one of the following:
      • member is < 13 years of age; or
      • member utilizes tube feeding (G-tube/J-tube); or
      • member has a swallowing disorder or condition affecting ability to swallow.

 

Zynteglo

  • Documentation of all of the following is required: 
    • diagnosis of transfusion-dependent beta thalassemia; and
    • copy of genetic test confirming diagnosis; and
    • prescriber is a hematologist or consult notes from a hematologist are provided; and
    • member is < 51 years of age; and
    • appropriate dosing and treatment dates; and
    • member does not have pre-existing HIV, HBV, or HCV infection; and
    • member has required ≥ 100 mL/kg/year of pRBC or ≥ eight transfusions within the last 12 months; and
    • infusion will take place in a qualified treatment center; and
    • member is clinically stable and eligible for HSCT.

 

Original Effective Date: 09/2003

Last Revised Date: 04/2024

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Last updated 04/01/24

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