IBM Micromedex® Compendium

Through the process established by the Centers for Medicare and Medicaid Services (CMS), IBM® Micromedex® DRUGDEX® is recognized by CMS as a compendium for the determination of payment for medically-accepted off-label uses for anti-cancer regimens. Effective January 1, 2010, new requirements were implemented that include a provision for providing transparency of processes used to evaluate therapies and to identify potential conflicts of interest. Specifically, publishers of compendia must now make the following information available via their public websites:

• Criteria used to evaluate the request (therapy)
• Disclosure of evidence considered
• Meeting minutes and records of votes for disposition of the request (therapy)
• Names of individuals who have substantively participated in the development of the compendia recommendations and disclosure of any potential conflicts of interest

To meet these requirements, documents detailing this information for each drug/off-label use pair published on or after January 1, 2010 are posted below. Information is organized by drug name, with the document title indicating the off-label use. Further transparency regarding Micromedex Off-Label processes can be found in the Micromedex Compendia Uses Policy.

DrugDex Off-Label Use Tracking

Abemaciclib

• Breast cancer; Early, hormone receptor-positive, HER2-negative, node-positive, high-risk, in combination with adjuvant endocrine therapy

Abiraterone Acetate

• Hormone sensitive prostate cancer, newly diagnosed, Metastatic, high-risk, in combination with prednisone and androgen-deprivation therapy

Acalabrutinib

• Waldenström macroglobulinemia

Acitretin

• Prophylaxis of skin cancers, in high-risk renal transplant recipients

Ado-Trastuzumab Emtansine

• Breast cancer; Advanced or metastatic, HER2-positive, first-line therapy
• Non-small cell lung cancer; Advanced disease, previously treated, HER2 mutation-positive

Afatinib Dimaleate

• Squamous cell carcinoma of the head and neck, Recurrent and/or metastatic disease, second-line as monotherapy after failure of platinum-based therapy

Alemtuzumab

• Chronic lymphocytic leukemia
• Graft versus host disease, in patients receiving allogeneic stem cell transplant for hematologic malignancies, steroid-refractory
• (PEDIATRIC) Graft versus host disease, in patients receiving allogeneic stem cell transplant for hematologic malignancies, steroid-refractory
• Primary cutaneous T‐cell lymphoma, Relapsed or refractory

Anastrozole

• Intraductal carcinoma in situ of breast, as adjuvant therapy in postmenopausal women with hormone receptor-positive disease
• Prevention of breast cancer in postmenopausal women at high risk
• Malignant tumor of ovary; Recurrent, estrogen- or progesterone-receptor positive, in postmenopausal women

Antithymocyte Globulin Equine

• Myelodysplastic syndrome

Antithymocyte Globulin Rabbit

• Graft versus host disease, prophylaxis in patients receiving unrelated donor hematopoietic stem cell transplantation for hematologic malignancies

Apixaban

• Thromboembolism of vein; Malignant neoplastic disease
• Thromboembolism of vein, Prophylaxis; In high-risk outpatients, Malignant neoplastic disease

Arsenic Trioxide

• Acute promyelocytic leukemia, FAB M3 newly diagnosed, low to intermediate risk, in combination with all-trans retinoic acid

Asparaginase Escherichia Coli

• Extranodal NK/T-cell lymphoma, nasal type

Atezolizumab

• Non-small cell lung cancer, advanced disease, 2nd or 3rd line after failure of platinum-based chemotherapy

Axitinib

• Metastatic renal cell carcinoma, first line therapy
• Renal cell carcinoma, Adjuvant therapy following nephrectomy in patients at high risk for recurrence

Bendamustine

• Mantle cell lymphoma; Previously untreated, transplant ineligible, in combination with rituximab
• Metastatic breast cancer
• Waldenström macroglobulinemia; In combination with rituximab

Bevacizumab

• Cancer of cervix, recurrent, persistent, or metastatic
• Colon cancer, adjuvant therapy in combination with fluorouracil, leucovorin, and oxaliplatin
• Extensive stage small cell lung cancer, First-line, in combination with chemotherapy
• Gastric cancer, advanced, first-line therapy in combination with fluoropyrimidine-based chemotherapy
• Glioblastoma multiforme of brain, newly diagnosed
• Liver carcinoma, advanced
• Mesothelioma
• Metastatic breast cancer, HER2-negative, first-line therapy in combination with chemotherapy (except paclitaxel)
• Metastatic breast cancer, HER2-negative, as second-line therapy in combination with other chemotherapy
• Necrosis of central nervous system due to exposure to ionizing radiation
• Nonsquamous non-small cell lung cancer, advanced or recurrent, first-line therapy in combination with cisplatin and gemcitabine
• Nonsquamous nonsmall cell neoplasm of lung; Stage IIIB/IV, continuation maintenance therapy as a single-agent following platinum-based, first-line therapy
• Nonsquamous non-small cell neoplasm of lung Stage IIIB/IV, first-line therapy in combination with pemetrexed and carboplatin
• Nonsquamous non-small cell neoplasm of lung, stage IIIB/IV or recurrent disease with EGFR exon 19 deletion or exon 21 mutation, first-line therapy in combination with erlotinib
• Ovarian cancer, advanced, first-line therapy in combination with carboplatin and paclitaxel

Bortezomib

• Amyloid light chain amyloidosis

Brentuximab vedotin

• Hodgkin lymphoma, consolidation therapy after autologous stem-cell transplantation
• Hodgkin’s disease; Relapsed or refractory (PEDIATRIC)

Cabozantinib Malate

• Renal cell carcinoma, Advanced, after failure of prior antiangiogenic therapy

Capecitabine

• Gastric cancer, stage II to IIIB, adjuvant therapy in combination with oxaliplatin
• Malignant tumor of breast; Early, HER2-negative, postoperative monotherapy in patients with residual disease after neoadjuvant anthracycline/taxane-based treatment
• Metastatic colorectal cancer, maintenance therapy following oxaliplatin-based induction chemotherapy in previously untreated patients
• Rectal cancer, locally advanced, adjuvant or neoadjuvant therapy, in combination with radiotherapy
• Stage III colon cancer, adjuvant, in combination with oxaliplatin
• Triple-negative breast cancer; Early, adjuvant therapy

Carboplatin

• Ovarian cancer, early-stage epithelial, adjuvant therapy
• Metastatic breast cancer, HER2 overexpression, first-line therapy in combination with a taxane and trastuzumab
• Testicular seminoma, stage I, adjuvant, monotherapy
• Triple negative breast cancer, neoadjuvant, in combination with chemotherapy

Carfilzomib

• Multiple myeloma; Newly diagnosed, transplant-eligible, in combination with an immunomodulatory drug and a steroid
• Multiple myeloma; Newly diagnosed, transplant-ineligible, in combination with a chemotherapy agent and a steroid
• Waldenstrom macroglobulinemia

Celecoxib

• Prevention of sporadic colorectal adenomas, in high-risk patients

Cetuximab

• Gastric or gastroesophageal junction cancer, advanced, as first-line therapy in combination with fluoropyrimidine-based chemotherapy

Cisplatin

• Malignant tumor of thyroid gland, Advanced, in combination with doxorubicin
• Malignant tumor of urinary bladder, Muscle invasive, as neoadjuvant combination chemotherapy
• Non-Hodgkin lymphoma, Relapsed or refractory, as part of the DHAP or ESHAP chemotherapy regimen
• Pancreatic cancer, locally advanced or metastatic, first-line therapy in combination with gemcitabine
• Triple negative breast cancer

Cladribine

• Acute myeloid leukemia, disease; Induction therapy
• Systemic mastocytosis

Cyclophosphamide

• Graft versus host disease, Prophylaxis, As monotherapy in patients who have received matched related or unrelated bone marrow transplantation for high risk hematologic malignancies with a myeloablative conditioning regimen

Dabrafenib Mesylate

• Malignant melanoma, Adjuvant, following complete resection of Stage III (with lymph node involvement greater than 1 mm) disease with BRAF V600E or V600K mutation, in combination with trametinib
• Non-small cell lung cancer, metastatic with BRAF V600E mutation, in prviously treated patients as monotherapy or in combination with trametinib

Dalteparin

• Thromboembolism of vein – Prophylaxis; In high-risk outpatients, Malignant neoplastic disease

Daratumumab

• AL amyloidosis – Relapsed or refractory

Dasatinib

• Chronic phase chronic myeloid leukemia, Philadelphia chromosome-positive, newly diagnosed
• Philadelphia chromosome-positive acute lymphoblastic leukemia; Newly diagnosed, in combination with chemotherapy

Defibrotide Sodium

• Condition Qualifier: Prophylaxis, ADULT, Veno-occlusive disease of the liver in patients undergoing hematopoietic stem cell transplantation
• Condition Qualifier: Prophylaxis, PEDIATRIC, Veno-occlusive disease of the liver in patients undergoing hematopoietic stem cell transplantation

Dexmethylphenidate

• Fatigue, cancer-related

Docetaxel

• Non-small cell lung cancer, advanced or metastatic, switch-therapy after gemcitabine/carboplatin
• Hormone sensitive prostate cancer, Metastatic, in combination with androgen-deprivation therapy

Doxorubicin Hydrochloride Liposome

• Mycosis fungoides, advanced or refractory, after failure of previous treatments
• Malignant tumor of ovary, first-line therapy, in combination therapy

Dutasteride

• Prostate cancer prophylaxis in high risk men

Edoxaban

• Thromboembolism of vein, Following parenteral therapy; in Malignant neoplastic disease

Eltrombopag Olamine

• Thrombocytopenic disorder, Monotherapy, Myelodysplastic syndrome (clinical)

Enoxaparin Sodium

• Treatment of venous thromboembolism in patients with cancer

Eribulin Mesylate

• Metastatic breast cancer; HER2-negative, first- or second-line chemotherapy for metastatic disease

Everolimus

• Hodgkin’s disease (clinical), Relapsed or refractory, in patients who had failed or were ineligible for autologous hematopoietic stem cell transplant or after failure of a gemcitabine-, vinorelbine-, or vinblastine containing regimen
• Malignant tumor of breast, advanced, hormone receptor-negative, HER2 positive, in trastuzumab-containing regimens
• Waldenstrom macroglobulinemia, relapsed or refractory

Exemestane

• Breast cancer, adjuvant, premenopausal, in combination with ovarian suppression
• Breast cancer, Neoadjuvant therapy for hormone receptor-positive disease in postmenopausal women
• Prevention of invasive breast cancer in postmenopausal women at increased risk

Fam-Trastuzumab Deruxtecan

• Non-small cell lung cancer; Advanced disease, previously treated, HER2 mutation-positive

Febuxostat

• Increased uric acid level in patients receiving chemotherapy and at intermediate to high risk of tumor lysis syndrome, Prophylaxis

Fluorouracil

• Bladder cancer, in combination with cisplatin and radiotherapy

Fosaprepitant Dimeglumine

• Prophylaxis, chemotherapy-induced nausea and vomiting in patients receiving cisplatin-based chemotherapy; prophylaxis, radiation-induced nausea and vomiting

Fulvestrant

• Malignant tumor of breast Locally advanced or metastatic, First-line endocrine therapy in postmenopausal women with hormone receptor-positive disease

Gefitinib

• Head and neck cancer, Squamous cell, recurrent or metastatic, as single-agent therapy

Gemcitabine Hydrochloride

• Malignant tumor of nasopharynx; Locoregionally advanced disease, as induction therapy before chemoradiation
• Malignant tumor of nasopharynx; Recurrent or metastatic, first-line therapy in combination with cisplatin
• Mantle cell lymphoma, relapsed or refractory, as combination therapy
• Non-small cell lung cancer; Stage IIIB/IV, continuation maintenance therapy as a single agent following first-line induction therapy with cisplatin and gemcitabine
• Ovarian cancer, Advanced, first-line therapy in combination with paclitaxel and carboplatin
• Peripheral t-cell lymphoma

Goserelin acetate

• Post-chemotherapy ovarian failure prophylaxis, in patients with breast cancer receiving adjuvant anthracycline and cyclophosphamide-based therapy

Hydroxyurea

• Intracranial meningioma, recurrent after surgery and radiotherapy

Ibrutinib

• Hairy cell leukemia (clinical); Relapsed/Refractory, monotherapy

Imatinib Mesylate

• Acute lymphoblastic leukemia, Philadelphia chromosome positive, newly diagnosed, as part of combination therapy [pediatric]
• Diffuse cutaneous systemic sclerosis

Inotuzumab

• Pre B-cell acute lymphoblastic leukemia; Relapsed or refractory (PEDIATRIC)

Irinotecan Hydrochloride

• Malignant tumor of pancreas; Locally advanced or metastatic, second-line in combination with a fluoropyrimidine

Iron Dextran

• Prophylaxis, chemotherapy-induced nausea and vomiting in patients receiving cisplatin-based chemotherapy; prophylaxis, radiation-induced nausea and vomiting

Isotretinoin

• OL Use: Neuroblastoma, high-risk, newly diagnosed, as post-consolidation therapy

Ixazomib

• Multiple myeloma; Newly diagnosed, maintenance therapy
• Waldenström macroglobulinemia; In combination with rituximab and dexamethasone

Ketamine Hydrochloride

• Cancer pain, Opioid therapy; Adjunct

Lenalidomide

• Myelofibrosis
• Myelodysplastic syndrome, transfusion-dependent anemia in patients at low –r intermediate-1 risk without  deletion 5q abnormality
• Non-Hodgkin lymphoma, in combination with rituximab
• POEMS syndrome
• Chronic lymphoid leukemia, disease; Maintenance, after chemotherapy
• AL amyloidosis; Relapsed or refractory, combination therapy

Melphalan Hydrochloride

• Retinoblastoma [Pediatrics]

Methylphenidate Hydrochloride

• Fatigue, cancer-related

Modafinil

• Fatigue, cancer-related

Naldemedine

• Opioid-induced constipation in patients with cancer

Nelarabine

• T-cell acute lymphoblastic leukemia; Newly diagnosed, intermediate- or high-risk disease, post-induction therapy in combination with the augmented Berlin-Frankfurt-Muenster (ABFM) regimen (PEDIATRIC)

Nilotinib

• Gastrointestinal stromal tumors, advanced, resistant to or intolerant of imatinib and sunitimib

Nilotinib Hydrochloride

• Philadelphia chromosome-positive acute lymphoblastic leukemia; Newly diagnosed, in combination with chemotherapy

Nintedanib

• Non-small cell lung cancer, Stage IIIB or IV disease, as second-line therapy in combination

Niraparib

• Malignant tumor of ovary, Fallopian tube, or primary peritoneal cancer, recurrent, platinum-sensitive disease, in combination with bevacizumab

Nivolumab

• Malignant melanoma, adjuvant, following complete resection of Stage IIIB, IIIC, or IV (resected distant metastases) disease
• Malignant mesothelioma of pleura; Progressive or recurrent disease following platinum-based chemotherapy
• Non-small cell lung cancer, metastatic or recurrent, PD-L1 expression, first-line treatment, with no EGFR or ALK tumor aberrations

Ofatumumab

• Waldenström macroglobulinemia

Olanzapine

• Chemotherapy-induced nausea and vomiting, moderately and/or highly emetogenic chemotherapy; Prophylaxis

Olaparib

• Malignant tumor of breast; Early, HER2-negative, germline BRCA mutation-positive, high-risk, after (neo)adjuvant chemotherapy and local treatment (radiation) therapy
• Malignant tumor of ovary, recurrent, platinum-sensitive, with at least 2 prior platinum-based chemotherapy regimens, as maintenance therapy
• Malignant tumor of pancreas; Metastatic, deleterious or suspected deleterious germline BRCA1 or BRCA2 mutation, as maintenance therapy in patients who did not progress during first-line platinum-based chemotherapy
• Metastatic breast cancer, HER2-negative, germline BRCA mutation-positive therapy
• Metastatic prostate cancer; Castration-resistant, previously treated with chemotherapy

Oxaliplatin

• Advanced or metastatic biliary tract cancer, in combination with gemcitabine
• Pancreatic cancer, advanced or metastatic, second-line therapy in combination with 5-fluorouracil and leucovorin
• Mantle cell lymphoma; As combination therapy
• Metastatic pancreatic adenocarcinoma, first-line treatment, in combination with 5-fluorouracil, leucovorin, and irinotecan

Paclitaxel protein-bound

• Malignant tumor of ovary, recurrent, platinum-resistant
• Malignant tumor of stomach, Refractory to first line fluoropyrimidine-containing chemotherapy
• Malignant tumor of breast; Neoadjuvant, in sequential combination with an anthracycline and cyclophosphamide

Palbociclib

• Malignant tumor of breast; Early, HER2-negative, hormone receptor-positive, in combination with adjuvant endocrine therapy

Panitumumab

• Metastatic colorectal cancer, wild-type KRAS mutation, first-line therapy, in combination with infusional fluorouracil, leucovorin, and oxaliplatin (FOLFOX4 regimen)
• Metastatic colorectal cancer, wild-type KRAS mutation, second-line therapy following fluoropyrimidine-containing chemotherapy, in combination with fluorouracil, leucovorin, and irinotecan (FOLFIRI regimen)
• Squamous cell carcinoma of head and neck

Panobinostat

• Multiple myeloma; Relapsed/refractory, in combination with carfilzomib

Pasireotide

• Carcinoid syndrome inadequately controlled with first generation somatostatin analogs, in patients with metastatic neuroendocrine tumors of the digestive tract

Pazopanib Hydrochloride

• Gastronintestinal stromal tumor, metastatic or advanced, after failure of imatinib and sunitinib
• Malignant tumor of ovary, Fallopian tube or primary peritoneal cancer, advanced, previously treated, in combination with paclitaxel
• Renal cell carcinoma, Adjuvant therapy following nephrectomy in patients at high risk for recurrence

Pegaspargase

• Extranodal NK/T-cell lymphoma, nasal type

Pegfilgrastim

• Harvesting of peripheral blood stem cells, prior to autologous stem-cell transplantation

Peginterferon alfa-2a

• Polycythemia vera
• Essential thrombocythemia

Peginterferon alfa-2b

• Essential thrombocythemia

Pembrolizumab

• Malignant mesothelioma of pleura; Previously treated
• Renal cell carcinoma; First-line, in combination with lenvatinib
• Triple-negative breast cancer; Early, neoadjuvant, in combination with standard chemotherapy

Pemetrexed

• Nonsquamous nonsmall cell neoplasm of lung; Stage IIIB/IV or recurrent, continuation maintenance therapy in combination with bevacizumab following platinum-based, first-line therapy
• Ovarian cancer, recurrent

Pertuzumab

• Malignant tumor of breast, adjuvant, HER2 overexpression, in combination with trastuzumab and chemotherapy
• Malignant tumor of stomach or Gastroesophageal junction cancer; Metastatic, HER2-positive, in combination with trastuzumab and chemotherapy

Pomalidomide

• AL amyloidosis; Relapsed or refractory, combination therapy with dexamethasone
• Multiple myeloma; Relapsed or refractory, in combination with a steroid in a triplet regimen

Ponatinib Hydrochloride

• Philadelphia chromosome-positive acute lymphoblastic leukemia; Newly diagnosed, in combination with chemotherapy

Porfimer Sodium

• Cholangiocarcinoma of the biliary tract, unresectable, after double stenting

Prochlorperazine

• Radiotherapy-induced nausea and vomiting, Prophylaxis

Pyridoxine

• Prophylaxis, acral erythema due to cytotoxic therapy in patients receiving capecitabine-containing chemotherapy

Ramucirumab

• Metastatic breast cancer, HER2-Negative, first-line, in combination with docetaxel
• Metastatic urothelial carcinoma; Or advanced, with progression after platinum-containing chemotherapy

Regorafenib

• Osteosarcoma of bone; Metastatic or advanced, progressive, previously treated

Risedronate Sodium

• Prophylaxis Osteopenia, Secondary to androgen-deprivation therapy in patients with prostate cancer

Rituximab

• Burkitt’s lymphoma (clinical) in combination with chemotherapy
• Chronic lymphoid leukemia, disease maintenance, following rituximab-containing chemotherapy
• Immune thrombocytopenia; In combination with a corticosteroid
• Immune thrombocytopenia, previously treated [pediatric patients]
• Mantle cell lymphoma, untreated, induction therapy, in combination with anthracycline-based regimens
• Mantle cell lymphoma untreated, maintenance therapy
• Post-transplant lymphoproliferative disorder [pediatric patients]
• Thrombotic thrombocytopenic purpura, in combination with plasma exchange
• Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukemia; CD20-positive, in combination with chemotherapy

Rivaroxaban

• Thromboembolism of vein; Malignant neoplastic disease
• Thromboembolism of vein, Prophylaxis; In high-risk outpatients, Malignant neoplastic disease

Romiplostim

• Chronic idiopathic thrombocytopenic purpura [pediatric]
• Thrombocytopenic disorder and Myelodysplastic syndrome

Ruxolitinib Phosphate

• Graft versus host disease following allogeneic stem call transpantation, steroid-refractory

Sodium Ferric Gluconate

• Prophylaxis, chemotherapy-induced nausea and vomiting in patients receiving cisplatin-based chemotherapy; prophylaxis, radiation-induced nausea and vomiting

Sorafenib Tosylate

• Acute myeloid leukemia, disease; FLT3-ITD mutation-positive, maintenance therapy following allogeneic HSCT
• Gastrointestinal stromal tumor Advanced or metastatic disease, after failing treatment with imatinib and sunitinib
• Malignant tumor of ovary; Primary peritoneal cancer, maintenance therapy
• Melanoma, unresectable or metastatic, in combination
• Non-small cell lung cancer, Advanced
• Renal cell carcinoma, Adjuvant therapy following nephrectomy in patients at high risk for recurrence

Sunitinib Malate

• Metastatic breast cancer, HER2-negative
• Non-small cell lung cancer, advanced or metastatic, in combination therapy, in previously treated patients
• Renal cell carcinoma, high-risk, adjuvant therapy

Tamoxifen

• Malignant tumor of breast, adjuvant therapy, premenopausal, hormone receptor-positive, in combination with ovarian suppression

Thalidomide

• Non-small cell lung cancer, advanced or metastatic, in combination with a platinum-based regimen
• Systemic mast cell disease

Trabectedin

• Malignant tumor of ovary; In combination with pegylated liposomal DOXOrubicin, following 1 or 2 previous platinum-based chemotherapy regimens

Trastuzumab

• Metastatic breast cancer HER2 overexpression, hormone receptor positive, postmenopausal women, in combination with anastrozole

Toremifene citrate

• Prophylaxis of vertebral fracture, Secondary to androgen deprivation therapy in patients with prostate cancer

Triptorelin pamoate

• Prevention of chemotherapy-induced early menopause, premenopausal women with breast cancer

Vandetanib

• Malignant tumor of ovary, recurrent, platinum-resistant
• Non-small cell lung cancer, locally advanced or metastatic, after failure of first- or second-line chemotherapy

Vedolizumab

• Acute graft-versus-host disease; Steroid-refractory

Vemurafenib

• Hairy cell leukemia (clinical); Relapsed/Refractory, in combination with rituximab
• Hairy cell leukemia (clinical); Relapsed/Refractory, monotherapy
• Malignant melanoma; Adjuvant therapy, cutaneous, with BRAF-V600 mutation

Vinorelbine

• Malignant mesothelioma of pleura
• Malignant pleural mesothelioma

Zanubrutinib

• Chronic lymphoid leukemia, disease; or Small lymphocytic lymphoma

Zoledronic acid

• Breast cancer, early stage, adjuvant therapy