Skip to Content

Table 78: Beta Thalassemia 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  

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, monitor complete blood count with WBC differential weekly and when blood counts have recovered, reinstitute hydroxyurea at 5 mg/kg/day and is 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 12 years of age and older.
    • FDA-approved dosing: 1,500 mg once daily.

 

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.

 

 Sickle Cell Disease Agents - Antimetabolites

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes

hydroxyurea Droxia test  
hydroxyurea Siklos test  

 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  
Table Footnotes

II. Therapeutic Uses

FDA-approved, for example:  

  • Beta thalassemia (Reblozyl)
  • Sickle cell disease (Adakveo, Endari, Oxbryta)

Note: The above list may not include all FDA-approved indications.

 

Back to top


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.
  • 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 the member’s condition and requested medication (see below).

   

Adakveo

  • Documentation of all of the following is required: 
    • appropriate diagnosis; and
    • member ≥ 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
    • an inadequate response to hydroxyurea at the maximally tolerated dose for at least three months; or
    • an adverse reaction or contraindication to hydroxyurea.
  • For recertification, documentation of postive response to therapy (e.g., decrease in VOCs, reduction in need for pain management, decrease in hospitalizations) is required.

 

Endari

  • Documentation of all of the following is required: 
    • appropriate diagnosis; and
    • member ≥ 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
    • an inadequate response, adverse reaction, or contraindication to hydroxyurea.

 

Reblozyl

  • Documentation of all of the following is required: 
    • medical records and genetic testing supporting diagnosis of transfusion-dependent beta thalassemia; and
    • member ≥ 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
    • appropriate dose.
  • For recertification, documentation of postive response to therapy (e.g., decrease in transfusion requirements) is required.

 

Oxbryta

  • Documentation of all of the following is required: 
    • appropriate diagnosis; and
    • member ≥ 12 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
    • 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
    • member has a baseline hemoglobin level ≤ 7 g/dL (Lab work should be drawn within the last 60 days).
  • For recertification, documentation of postive 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.

 


Original Effective Date: 02/2020

Last Revised Date: 04/2020


Clinical Criteria Main Page | Back to topPrevious  |  Next

Last updated 08/03/20

Feedback