Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Moderate to severe chronic graft versus host disease (cGVHD)

Initial treatment

Patient must have received prior systemic steroid treatment for this condition; AND

Patient must be one of the following: (i) refractory to steroid treatment, (ii) dependent on steroid treatment, (iii) intolerant to steroid treatment; AND

The treatment must be the sole PBS-subsidised treatment for this condition with the exception of: (i) corticosteroids, (ii) calcineurin inhibitors; AND

Must be treated by a haematologist; or

Must be treated by an oncologist with allogeneic bone marrow transplantation experience; or

Must be treated by a medical practitioner working under the direct supervision of one of the above mentioned specialist types; AND

Patient must be undergoing treatment with this drug following allogeneic haematopoietic stem cell transplantation.

The severity of cGVHD is defined by the National Institutes of Health (NIH) criteria (Jagasia et al., 2015):

(a) Moderate cGVHD: at least one organ (not lung) with a score of 2, 3 or more organs involved with a score of 1 in each organ, or lung score of 1

(b) Severe cGVHD: at least 1 organ with a score of 3, or lung score of 2 or 3

Steroid-refractory disease is defined as:

(a) a lack of response or disease progression after administration of a minimum prednisone dose of 1 mg/kg/day for at least 1 week (or equivalent); or

(b) disease persistence without improvement despite continued treatment with prednisone at greater than 0.5 mg/kg/day or 1 mg/kg/every other day for at least 4 weeks (or equivalent).

Steroid-dependent disease is defined as an increased prednisone dose to greater than 0.25 mg/kg/day after two unsuccessful attempts to taper the dose (or equivalent).

Steroid intolerance is defined as a patient developing an intolerance of a severity necessitating treatment withdrawal.

Details of prior steroid use should be documented in the patient's medical records.

A patient must demonstrate a response 24 weeks after initiating treatment with ruxolitinib to be eligible for continuing treatment.

Response is defined as attaining a complete or partial response as defined by the National Institutes of Health (NIH) criteria (Lee et al., 2015). Note that response is relative to the assessment of organ function affected by cGVHD prior to commencing initial treatment with ruxolitinib.

(a) complete response is defined as complete resolution of all signs and symptoms of cGVHD in all evaluable organs without initiation or addition of new systemic therapy.

(b) partial response is defined as an improvement in at least one organ (e.g. improvement of 1 or more points on a 4-to-7-point scale, or an improvement of 2 or more points on a 10-to-12-point scale) without progression in other organs or sites, initiation or addition of new systemic therapies.

The assessment of response must be documented in the patient's medical records.

This drug is not PBS-subsidised if it is prescribed to an in-patient in a public hospital setting.

Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Moderate to severe chronic graft versus host disease (cGVHD)

Continuing treatment

Patient must have received initial PBS-subsidised treatment with this drug for this condition; AND

Patient must have responding disease at 24 weeks compared with baseline, demonstrated by either a: (i) partial response, (ii) complete response; AND

The treatment must be the sole PBS-subsidised treatment for this condition with the exception of: (i) corticosteroids, (ii) calcineurin inhibitors; AND

Must be treated by a haematologist; or

Must be treated by an oncologist with allogeneic bone marrow transplantation experience; or

Must be treated by a medical practitioner working under the direct supervision of one of the above mentioned specialist types.

Response is defined as attaining a complete or partial response as defined by the National Institutes of Health (NIH) criteria (Lee et al., 2015). Note that response is relative to the assessment of organ function affected by cGVHD prior to commencing initial treatment with ruxolitinib.

(a) complete response is defined as complete resolution of all signs and symptoms of cGVHD in all evaluable organs without initiation or addition of new systemic therapy.

(b) partial response is defined as an improvement in at least one organ (e.g. improvement of 1 or more points on a 4-to-7-point scale, or an improvement of 2 or more points on a 10-to-12-point scale) without progression in other organs or sites, initiation or addition of new systemic therapies.

The assessment of response must be documented in the patient's medical records.

Tapering the dose of corticosteroids should be considered in patients with responding disease. Following successful tapering of corticosteroids, tapering the dose of ruxolitinib can be initiated.

This drug is not PBS-subsidised if it is prescribed to an in-patient in a public hospital setting.