CurrentCareSourcePolicy N/A

UTILIZATION MANAGEMENT MEDICAL POLICY

Defines prior authorization and coverage criteria for Grafapex (treosulfan intravenous infusion) in combination with fludarabine as a preparatory regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in adult and pediatric patients ≥ 1 year with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Approvals are time-limited and require specialist prescribing/consultation.

Policy Summary

PayerCareSource

PolicyUTILIZATION MANAGEMENT MEDICAL POLICY

Policy CodePolicy N/A

Change Typerevised

Effective DateFeb 26, 2025

Next Review Date

Key ActionPrior authorization is recommended; approvals provided for 30 days and Grafapex must be prescribed by or in consultation with a hematology/oncology/transplant specialist.

SourceLink

POLICY UPDATE CHANGES

New policy/review date recorded 02/26/2025 with inclusion of Grafapex (treosulfan) indications, dosing, and prior authorization criteria.

2Covered Indications (AML, MDS)

30 daysStandard Approval Duration

≥1Minimum Age (years)

Coverage Summary

Defines prior authorization and coverage criteria for Grafapex (treosulfan intravenous infusion) in combination with fludarabine as a preparatory regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in adult and pediatric patients aged ≥ 1 year with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

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Medical Necessity / Authorization Criteria

FDA-Approved Indications - Acute Myeloid Leukemia (AML)

Approve for 30 days if the patient meets ALL of the following (A, B, C, and D):

ALL of the following

A: Patient is ≥ 1 year of age
B: Grafapex is being used in combination with fludarabine
C: Patient is undergoing allogeneic hematopoietic stem cell transplantation
D: Medication is prescribed by or in consultation with a hematologist, oncologist, transplant specialist physician, or a physician associated with a transplant center

ref': 'b1_0

title': 'FDA-Approved Indications - Acute Myeloid Leukemia (AML)'

PLACEHOLDER

Dosing for AML

Approve dosing when the following dosing limits are respected:

ALL of the following

Dose up to 10 g/m2 body surface area per dayUp to 10 g/m2 body surface area per day
Administered as a 2-hour intravenous infusion
Given for up to 3 consecutive daysUp to 3 consecutive days

FDA-Approved Indications - Myelodysplastic Syndrome (MDS)

Approve for 30 days if the patient meets ALL of the following (A, B, C, and D):

ALL of the following

A: Patient is ≥ 1 year of age
B: Grafapex is being used in combination with fludarabine
C: Patient is undergoing allogeneic hematopoietic stem cell transplantation
D: Medication is prescribed by or in consultation with a hematologist, oncologist, transplant specialist physician, or a physician associated with a transplant center

Dosing for MDS

Approve dosing when the following dosing limits are respected:

ALL of the following

Dose up to 10 g/m2 body surface area per dayUp to 10 g/m2 body surface area per day
Administered as a 2-hour intravenous infusion
Given for up to 3 consecutive daysUp to 3 consecutive days

Conditions Not Recommended for Approval

Coverage is not recommended when the request does not meet the Recommended Authorization Criteria:

Any circumstance not listed in the Recommended Authorization Criteria

Provider Actions / Authorization Requirements

Prior Authorization

Prior authorization required

Prior authorization is recommended for medical benefit coverage of Grafapex. Approvals are provided for 30 days. Requests for doses outside the documented dosing will be considered on a case-by-case basis by a clinician (Medical Director or Pharmacist).

Documentation Required

Specialist prescribing/consultation required

Grafapex must be prescribed by or in consultation with a hematologist, oncologist, transplant specialist physician, or a physician associated with a transplant center.

Billing Rule

Dosing administration note

Administered as a 2-hour intravenous infusion. Dosing limits: up to 10 g/m2 body surface area per day, given for up to 3 consecutive days. Requests for other dosing or schedules will be reviewed case-by-case.

Background & Evidence

Grafapex is an alkylating agent (treosulfan) indicated in combination with fludarabine as a conditioning regimen for allogeneic HSCT in adult and pediatric patients aged ≥ 1 year for the treatment of AML and MDS.

Conditioning regimens are used in allogeneic HSCT to eradicate malignant marrow cells (for myeloablative regimens) and to immunosuppress the recipient to promote donor engraftment.

Recommended dosing is up to 10 g/m2 per day administered as a 2-hour intravenous infusion for up to 3 consecutive days when used as part of the transplant preparative regimen.

Grafapex carries a Boxed Warning for myelosuppression and requires specialist prescribing/consultation; prior authorization is recommended and approvals are provided for 30 days to allow administration of the regimen.

Source	Notes
NCCN guidance	NCCN HCT guidelines (v2.2024) have not addressed Grafapex as of Aug 30, 2024
Prescribing information	Grafapex prescribing information, Medexus; January 2025

Grafapex = Brand name for treosulfan intravenous infusion (Medexus).

Treosulfan = Alkylating agent used as part of conditioning regimens for allogeneic HSCT.

Conditioning regimen = Preparatory treatment to eradicate malignant marrow cells and immunosuppress the recipient to promote donor engraftment prior to allogeneic HSCT.

Specialist prescriber = Hematologist, oncologist, transplant specialist physician, or a physician associated with a transplant center (required for approval).

OpenPayer

UTILIZATION MANAGEMENT MEDICAL POLICY

Coverage Summary

Medical Necessity / Authorization Criteria

Coding

Provider Actions / Authorization Requirements

Background & Evidence

Revision History