Facts and figures of the compassionate use of onco-hematological drugs from 2016 to 2021: Results from the multicentric observational study Compass-O.

Background: Compassionate drug use (CDU) is a topic of debate in the scientific community characterized by a lack of information, especially in the onco-hematology area. Aim: The Compass-O study aimed to provide updated data on CDU of onco-hematological drugs for the period 2016–2021. Method: Compas...

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Publicado en:Journal of Oncology Pharmacy Practice Vol. 32; no. 6; pp. 1008 - 1016
Autores principales: Mascolo, Annamaria, Dell'Anno, Irene, Dondi, Leonardo, Scavone, Cristina, Capuano, Annalisa, Giordano, Marialuisa, Cristinziano, Adriano, Tarantino, Domenico, Pani, Marcello, Masini, Carla, Donati, Caterina, Rossin, Elisabetta, Lagana, Graziella, Ballerio, Alice, Bagaglini, Gabriele, Bonanni, Gabriella, Vergati, Alberto, Cavaliere, Arturo, Celenza, Rosanna, D'Arpino, Alessandro
Formato: research tables/charts Journal Article
Publicado: Sage Publications Inc. Sep2026
Acceso en línea:Ver este registro en EBSCOhost
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      dt: Sep2026
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      pub: Sage Publications Inc.
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        atl: Facts and figures of the compassionate use of onco-hematological drugs from 2016 to 2021: Results from the multicentric observational study Compass-O.
      aug:
        au:
          Mascolo, Annamaria
          Dell'Anno, Irene
          Dondi, Leonardo
          Scavone, Cristina
          Capuano, Annalisa
          Giordano, Marialuisa
          Cristinziano, Adriano
          Tarantino, Domenico
          Pani, Marcello
          Masini, Carla
          Donati, Caterina
          Rossin, Elisabetta
          Lagana, Graziella
          Ballerio, Alice
          Bagaglini, Gabriele
          Bonanni, Gabriella
          Vergati, Alberto
          Cavaliere, Arturo
          Celenza, Rosanna
          D'Arpino, Alessandro
        affil: Department of Experimental Medicine, Section of Pharmacology "L. Donatelli", University of Campania "Luigi Vanvitelli", Napoli, Italy
      sug:
        subj:
          Hematologic Diseases Drug Therapy
          Neoplasms Drug Therapy
          Antineoplastic Agents Therapeutic Use
          Drugs, Off-Label
          Drug Utilization Trends
          Human
          Male
          Multicenter Studies
          Female
          Nonexperimental Studies
          Retrospective Design
          Record Review
          Italy
          Lung Neoplasms Drug Therapy
          Nivolumab Therapeutic Use
          Leukemia, Myeloid, Acute Drug Therapy
          Azacitidine Therapeutic Use
          Breast Neoplasms
          Leukemia, Lymphocytic, Chronic
          Multiple Myeloma
          Conceptual Framework
          Descriptive Statistics
          Cost Savings
          Insurance, Health, Reimbursement
          Male
          Female
      ab: Background: Compassionate drug use (CDU) is a topic of debate in the scientific community characterized by a lack of information, especially in the onco-hematology area. Aim: The Compass-O study aimed to provide updated data on CDU of onco-hematological drugs for the period 2016–2021. Method: Compass-O is a retrospective observational study with data obtained from 7 Italian Units for cytotoxic drug preparations (Unità Farmaci Antiblastici, UFA). Drugs and Therapeutic Indications (TI) were described separately and as drug-TI combinations, providing the mean Patients Per Year (mPPY) and the Years of Use (YoU). Results: A total of 783 requests of CDU were retrieved, referring to 156.3 mPPY (max: 272 in 2021; min: 84 in 2018). The 52.2% of subjects was female, and the 63.2% aged >60 years. A total of 709 (90.5%) CDUs referred to solid tumors and 74 (9.5%) to liquid tumors. CDU referred to 93 drug-TI combinations. The most recurrent drug-TIs were nivolumab-lung carcinoma (mPPY: 36.5; YoU: 2) for solid tumors, and azacytidine-acute myeloid leukemia (6.0; 1) for liquid tumors. The most frequent TIs were lung carcinoma (45.7; 6) and breast cancer (37.0; 4) for solid tumors, and chronic lymphocytic leukemia (5.0; 2) and multiple myeloma (4.7; 6) for liquid tumors. Conclusion: Compass-O provided an updated framework of the CDU in onco-hematology, showing its large-scale phenomenon and the relevance of UFA as a data source, underlining the need for a national observatory to monitor CDU.
      pubtype: Academic Journal
      doctype:
        research
        tables/charts
        Journal Article
      ougenre: Article
    language: English
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