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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.

 

Beta thalassemia and myelodysplastic syndromes (MDS)

  • Betibeglogene autotemcel is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of adult and pediatric patients with beta 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.
  • Imetelstat is an oligonucleotide telomerase inhibitor that blocks the interaction between telomerase and telomeres, leading to the increased destruction of malignant cells with high telomerase activity. This inhibition can improve hematopoiesis in the bone marrow. Imetelstat is currently indicated for adults with low- to intermediate-1 risk MDS with transfusion-dependent anemia requiring ≥ four RBC units over eight weeks who have not responded to, or have lost response to, or are ineligible for, erythropoiesis stimulating agents (ESA).
  • Luspatercept-aamt is a subcutaneously (SC) administered erythroid maturation agent.
    • This agent is FDA-approved for:
      • the treatment of anemia in adults with beta thalassemia who require regular RBC transfusions
      • the treatment of anemia without previous ESA use in adults with very low- to intermediate-risk MDS who may require regular RBC transfusions
      • the treatment of anemia failing an ESA and requiring ≥ two RBC units over eight weeks in adults with very low- to intermediate-risk MDS with ring sideroblasts (RS) or with MD/myeloproliferative neoplasm with RS and thrombocytosis (MDS/MPN-RS-T)
    • 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.

 

Sickle Cell Disease (SCD)

  • Crizanlizumab-tmca is the first humanized anti-P-selectin monoclonal antibody FDA-approved to reduce the frequency of vaso-occlusive crises (VOCs) in adults and pediatric patients aged 16 years and older with sickle cell disease.
    • This agent should be administered by a medical professional.
  • Exagamglogene autotemcel is an autologous, ex vivo CRISPR/Cas9 gene-editing therapy indicated for the treatment of patients 12 years of age and older with SCD with recurrent VOCs and for the treatment of transfusion-dependent beta thalassemia (TDT). This agent is a one-time IV infusion that works to edit the erythroid-specific enhancer region of BCL11A in the CD34hematopoietic 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 autotemcel, please see the Acute Hospital Carve-Out Drugs List found at www.mass.gov/druglist.
  • Hydroxyurea (HU) is available in several formulations, tablets and capsules. It is indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in patients with sickle cell anemia with recurrent moderate to severe painful crises.
    • Guidelines from the British Society for Haematology and the National Heart, Lung, and Blood Institute (NHLBI) recommend the use of HU 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 HU and monitor complete blood count with WBC differential weekly. When blood counts have recovered, reinstitute HU 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 HU 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
  • L-glutamine is an oral agent indicated to reduce acute complications in children ≥ five years of age and adults with SCD.
  • Lovotibeglogene autotemcel is a one-time gene therapy treatment designed to add functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own HSCs, utilizing the BB305 lentiviral vector. Once patients have the βA-T87Q-globin gene, their RBCs can produce anti-sickling hemoglobin (HbAT87Q) that decreases the proportion of HbS, with the goal of reducing sickled RBCs, hemolysis, and other complications. This gene therapy is approved for the treatment of patients 12 years of age and older with SCD and a history of vaso-occlusive events. 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 lovotibeglogene autotemcel, 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 Casgevy PD PA   CO, MB
lovotibeglogene autotemcel Lyfgenia PA   CO, MB

 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

 Telomerase Inhibitor

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes

imetelstat Rytelo 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 (Casgevy, Reblozyl, Zynteglo)
  • Myelodysplastic syndromes associated anemia (Reblozyl, Rytelo)
  • Sickle cell disease (Adakveo, Casgevy, l-glutamine, Lyfgenia, 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 sickle cell crises in the previous 12 months; and
    • member's current weight; and
    • one of the following:
      • 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 for a diagnosis of sickle cell disease:                            
    • appropriate diagnosis; 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 will receive pre-infusion conditioning with busulfan; 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.
  • Documentation of all of the following is required for a diagnosis of transfusion-dependent beta thalassemia (TDT or beta thalassemia major):                            
    • appropriate diagnosis; and
    • copy of genetic test confirming diagnosis; and
    • prescriber is a hematologist or consult notes from a hematologist are provided; and
    • member is ≥ 12 years of age; and
    • appropriate dosing and treatment dates; and
    • member has required ≥ 100 mL/kg/year of pRBC or ≥ ten units per year in the previous two years; and
    • infusion will take place in a qualified treatment facility; and
    • member will receive pre-infusion conditioning with busulfan; 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 TDT gene therapy.

 

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

 

l-glutamine

  • 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 sickle cell crises in the previous 12 months; and
    • member's current weight; and
    • inadequate response, adverse reaction, or contraindication to hydroxyurea.

 

Lyfgenia

  • 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 
    • medical necessity for use of requested agent instead of Casgevy; and
    • member has a negative serology test for HIV; and
    • member does not have α-thalassemia trait (-α3.7/-α3.7); 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 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.

 

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 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 are provided; and
    • member's current weight.
  • For recertification, documentation of positive response to therapy (e.g., decrease in transfusion requirements) is required.

 

Rytelo

  • 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 are provided; and
    • appropriate dosing; and
    • member has required ≥ four RBC transfusions in the last eight weeks; and
    • inadequate response or adverse reaction to one or contraindication to all erythropoiesis stimulating agents (e.g., epoetin, darbepoetin); and
    • if the member has MDS with ring sideroblasts (RS), inadequate response, adverse reaction, or contraindication to Reblozyl; and
    • if the member has MDS associated with a del 5q cytogenic abnormality, inadequate response, adverse reaction, or contraindication to lenalidomide.
  • 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 has a negative serology test for HIV; 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; and
    • member has not received any prior gene therapy for transfusion-dependent beta thalassemia.


Original Effective Date: 09/2003

Last Revised Date: 01/2025


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Last updated 01/06/25

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