Sharon is a 70-year-old female who is diagnosed with acute myeloblastic leukemia (C92.00), with 39% blast. She also suffers from rheumatoid arthritis, DJD, hypertension, depression hypothyroidism and obesity. Sharon was previously treated with decitabine, but still experienced disease progression. Your denial is due to an off-label diagnosis stating as not medically necessary for Venclexta treatment. Venclexta has received an FDA breakthrough therapy designation for use in combination with low dose cytarabine (LDAC) in treatment-naïve elderly patients with acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. Venclexta is a highly selective, orally bioavailable BCL2 inhibitor that has shown activity in BCL2-dependent leukemia and lymphoma cell
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In a Phase II study Venclexta monotherapy demonstrated clinical activity in patients with AML (relapsed/refractory or unfit for intensive chemotherapy) with a tolerable safety profile. B-cell leukemia/lymphoma-2 (BCL2), an antiapoptotic protein commonly expressed in hematologic malignancies, has been shown to be involved in tumor survival and chemo-resistance. Venclexta induced cell death in AML cell lines and primary patient samples in vitro and in mouse xenograft models. BCL2 inhibition induced cell death in AML cell lines and in leukemic blasts, progenitor cells, and stem cells from patients with AML, such as in Sharon’s case. The unique role of BCL2 in leukemia stem cell survival suggests that BCL2 inhibition has the potential to eliminate chemotherapy-resistant leukemia stem cells while sparing normal hematopoietic stem cells. The use of drugs for indications beyond those stipulated in the FDA label has been a physician prerogative that the FDA has approved of for more than 30 years, therefore, Venclexta is her best treatment option. Sharon needs the best supportive care, for this reason I respectfully request Venclexta be approved for Sharon now so her therapy is no longer, unnecessarily
In a Phase II study Venclexta monotherapy demonstrated clinical activity in patients with AML (relapsed/refractory or unfit for intensive chemotherapy) with a tolerable safety profile. B-cell leukemia/lymphoma-2 (BCL2), an antiapoptotic protein commonly expressed in hematologic malignancies, has been shown to be involved in tumor survival and chemo-resistance. Venclexta induced cell death in AML cell lines and primary patient samples in vitro and in mouse xenograft models. BCL2 inhibition induced cell death in AML cell lines and in leukemic blasts, progenitor cells, and stem cells from patients with AML, such as in Sharon’s case. The unique role of BCL2 in leukemia stem cell survival suggests that BCL2 inhibition has the potential to eliminate chemotherapy-resistant leukemia stem cells while sparing normal hematopoietic stem cells. The use of drugs for indications beyond those stipulated in the FDA label has been a physician prerogative that the FDA has approved of for more than 30 years, therefore, Venclexta is her best treatment option. Sharon needs the best supportive care, for this reason I respectfully request Venclexta be approved for Sharon now so her therapy is no longer, unnecessarily