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Chapter 1, Molecular concepts in hematology and cellular basis of hematopoiesis
Berthold Göttgens: nothing to disclose.
Gabriel Ghiaur: nothing to disclose.
Chapter 2, Consultative hematology 1: hospitalbased and selected outpatient topics
Jennifer R. Green: honoraria: North American ThrombosisForum, Scripps Health. Board membership: EPIC Hematology Steering Board. Grant funding: Health Resources and Services Administration, Centers for Disease Control and Prevention, Patient Centered Outcomes Research Institute, American Thrombosis and Hemostasis Network.
Shannon L. Carpenter: Consultancy: CSL Behring, Kedrion Biopharmaceuticals, HEMA Biologics; Novo Nordisk Pharmaceuticals, Inc; Bayer; Genentech. Membership on board of directors: Hemostasis and Thrombosis Research Society. Grant funding: Shire, CSL Behring.
Chapter 3, Consultative hematology 2: women’s health issues
Leslie Skeith: research funding: CSL Behring. Honoraria: Leo Pharma, Sanofi.
Molly Weidner Mandernach: nothing to disclose.
Andra H. James: research funding: Coagulant Therapeutices. Consulting: Cerus, Octapharma, Tremeau, Hemosonics, Coagulant Therapeutics.
Chapter 4, Hematopoietic growth factors
Sophie Park: research support: Novartis, Pfizer, Takeda, Sandoz, and BMS for research in myelodysplastic syndromes.
Gerald A. Soff: research support: Amgen, Dova/Sobi Pharmaceuticals, and Janssen Scientific Affairs. Advisory boards (in the past 5 years: Amgen, Janssen Scientific Affairs, Bayer Pharmaceuticals, Dova Pharmaceuticals, Bristol Myers Squibb, Pfizer, Novartis, Anthos Therapeutics, and Hengrui (USA) Ltd.
Chapter 5, Iron physiology, iron overload, and the porphyrias
Elizabeta Nemeth: stock ownership: Intrinsic LifeSciences and Silarus Therapeutics. Consultancy: Protagonist Therapeutics, Ionis Pharmaceuticals, Disc Medicine, and Astra- Zeneca-Fibrogen.
Carol Mathew: nothing to disclose.
Chapter 6, Acquired underproduction anemias
Jacquelyn M. Powers: research funding to her institution: American Regent.
Maria Domenica Cappellini: nothing to disclose
Chapter 7, Thalassemia, sickle cell disease, and other hemoglobinopathies
Payal Desai: advisory Board: Pfizer, GBT, Takeda, and Forma. Speaker & Research Project: Novartis.
Rakhi Naik: Rigel, Elsevier.
Jeffrey Lebensburger: consultant: Novartis, Emmaus, Forma Therapeutics, Agios.
Chapter 8, Hemolytic anemias excluding hemoglobinopathies Kevin H. M. Kuo: consultancy: Agios, Alexion, Apellis, Aruvant, Bioverativ/Sanofi, Bluebirdbio, Celgene/BMS, Forma, Novartis, and Pfizer. Member of the scientific advisory board: Agios. Honoraria: Agios, Alexion. and Novartis. Research support: Pfizer. Scientific collaboration: Abfero and Phoenicia Biosciences.
Samuel A. Merrill: member of scientific advisory board: True North Therapeutics, Alexion, and Rigel.
Chapter 9, Thrombosis and thrombophilia
Paul Monagle: nothing to disclose.
Bethany Samuelson Bannow: nothing to disclose.
Chapter 10, Bleeding disorders
Sven Olson: nothing to disclose.
Riten Kumar: consultancy: Bayer.
Chapter 11, Disorders of platelet number and function
Hanny Al-Samkari: consultancy: Agios, Dova, Argenx, Sobi, and Rigel. Research funding: Amgen, Agios, and Dova.
Michele P. Lambert: advisory board membership: Octapharma and Shionogi. Consultancy: Amgen, Novartis, Shionogi, Dova, Principia, Argenx, Rigel, and Bayer. Research funding: Sysmex, Novartis, Rigel, and AstraZeneca.
Chapter 12, Laboratory hematology
Kristi J. Smock: nothing to disclose.
Kenneth D. Friedman: consultancy: Instrumentation Laboratory, Siemens, and Takeda.
Chapter 13, Transfusion medicine
Michelle P. Zeller: medical officer with Canadian Blood Services. Honoraria: Pharmacosmos, Pfizer, and American Society of Hematology; research funding: Canadian Institute of Health Research, Canadian Blood Services, and Pfizer Global Medical Grants.
Stella T. Chou: consultancy: Platelet-Bio. Honoraria: American Society of Hematology, Wolters Kluwer, and Engage Health Media. Research funding: National Institutes of Health and Hyundai Hope on Wheels.
Chapter 14, Hematopoietic cell transplantation and cellular therapy
Irum Khan: honoraria: Takeda and Celgene.
Nirav N. Shah: honoraria and/or travel support: Incyte, Celgene, Lily, and Miltenyi Biotec. Membership on scientific advisory board: Lilly, Kite, Celgene, Legend, Epizyme, Seattle Genetics, and TG Therapeutics. Equity ownership in: Exelixis and Geron. Institutional research support for clinical trials: Miltenyi Biotec.
Matthew Ulrickson: nothing to disclose.
Chapter 15, Myeloid disorders and inherited bone marrow failure syndromes
Boglarka Gyurkocza: clinical trial support: Actinium Pharmaceuticals.
Marcin Wlodarski: nothing to disclose
Chapter 16, Chronic myeloid leukemia
Kendra L. Sweet: research funding: Incyte. Membership on advisory boards: Novartis, Bristol Myers Squibb and Arog. Honorarium: Astellas.
Ehab L. Atallah: research funding: Novartis and Takeda. Consultancy: Pfizer, Celgene, Novartis, and Takeda.
Chapter 17, Myeloproliferative neoplasms
Angela Fleischman: membership on advisory board: PharmaEssentia, CTI, and BMS. Kristen Pettit: membership on an advisory board: PharmaEssentia and Kura Oncology.
Chapter 18, Acquired marrow failure syndromes: aplastic anemia, paroxysmal nocturnal hemoglobinuria, and myelodysplastic syndromes
Bhumika J. Patel: speaker bureau: Alexion and Apellis.
Catherine Smith: research funding: AbbVie. Clinical trial: Celgene.
Chapter 19, Acute myeloid leukemia
Neerav Shukla: nothing to disclose.
Michael F. Walsh: nothing to disclose.
Roland B. Walter: laboratory research grants and/or clinical trial support: Amgen, Aptevo, Celgene, ImmunoGen, Janssen, Jazz, MacroGenics, Pfizer, and Selvita. Ownership interest in: Amphivena. Consultancy: Amphivena, Astellas, Bristol Myers Squibb, Genentech, GlaxoSmithKline, Janssen, Kite, Kronos, and MacroGenics.
Chapter 20, Acute lymphoblastic leukemia and lymphoblastic lymphoma
Oliver G. Ottmann: research funding: Incyte and Amgen.
Liora Schultz: nothing to disclose.
Chapter 21, Hodgkin lymphoma
Celeste Bello: research advisory board (with honorarium): Seattle Genetics.
Pamela B. Allen: research advisory board (with honorarium): Bayer, Daichii Sankyo, Imbrium, and Kyowa Kirin. Research funding: Kyowa Kirin.
Chapter 22, Indolent non-Hodgkin lymphomas
Sairah Ahmed: nothing to disclose.
David T. Yang: consultancy: Novartis.
Chapter 23, Aggressive non-Hodgkin and Burkitt lymphoma
Allison Rosenthal: nothing to disclose.
Amitkumar Mehta: research funding: Incyte, Takeda, Forty Seven Inc/Gilead, Juno Pharmaceuticals/BMS, Celgene/BMS, OncoTartis, Innate Pharmaceuticals, SeaGen, TG Therapeutics, Affimed, Merck, Kite/Gilead, Roche-Genentech, and ADC Therapeutics. Consultancy, speaker, or advisory board member: Gilead, AstraZeneca, Pharmacyclics, SeaGen, Morphosys/Incyte, TG Therapeutics, Kyowa Kirin, and Rigel Pharmaceuticals.
Chapter 24, Chronic lymphocytic leukemia/small lymphocytic lymphoma
Tanya Siddiqi: speaker: Pharmacyclics, AstraZeneca, Bristol Myers Squibb, and Janssen. Advisor: Pharmacyclics, Astra- Zeneca, Bristol Myers Squibb, Juno Therapeutics, Celgene, Kite Pharma, and BeiGene.
Jennifer Woyach: consultant: Pharmacyclics, Janssen, AbbVie, AstraZeneca, BeiGene, Arqule, and Loxo.
Chapter 25, Plasma cell disorders Caitlin Costello: consultancy: BMS, Janssen, Takeda, Karyopharm, and Oncopeptides. Research funding: Celgene, Takeda, Pfizer, and Poseida.
Rafael Fonseca: consultancy: Amgen, BMS, Celgene, Takeda, Bayer, Janssen, Novartis, Pharmacyclics, Sanofi, Karyopharm, Merck, Juno, Kite, Aduro, OncoTracker, Oncopeptides, GSK, and AbbVie. Scientific advisory board member: Adaptive Biotechnologies, Caris Life Sciences, and OncoTracker.
Chapter 1, Molecular concepts in hematology and cellular basis of hematopoiesis
No discussion of off-label drug use.
Chapter 2, Consultative hematology 1: hospital based and selected outpatient topics
Jennifer R. Green and Shannon L. Carpenter: corticosteroids and rituximab for use in TTP; ATIII and activated protein C for DIC; cyclophosphamide, corticosteroids, IVIG, and rituximab for CAPS; corticosteroids, intravenous immunoglobulin, rituximab, and thrombopoietin receptor agonists for pediatric ITP; rituximab for adult ITP and TTP; granulocyte-stimulating factor outside of severe congenital neutropenia. Desmopressin for use in platelet function disorders; desmopressin, recombinant FVIIa, prothrombin complex concentrate, activated prothrombin complex concentrate, fibrin glue, ε-aminocaproic acid, and tranexamic acid for use in surgical bleeding and reversal of direct oral anticoagulants; IVIG for posttransfusion purpura; IVIG for drug-induced ITP; conjugated estrogens for use in uremic bleeding; hemoglobin-based oxygen carriers for anemia in patients who refuse blood products.
Chapter 3, Consultative hematology 2: women’s health issues
Almost all medications are considered off-label in pregnancy.
Chapter 4, Hematopoietic growth factors
Sophie Park: deferasirox and lenalidomide in myelodysplastic syndromes.
Gerald A. Soff: all discussion of thrombopoietin-related drugs is currently off-label.
Chapter 5, Iron physiology, iron overload, and the porphyrias
Off-label use of iron chelation therapy.
Chapter 6, Acquired underproduction anemias
No discussion of off-label drug use.
Chapter 7, Thalassemia, sickle cell disease, and other hemoglobinopathies
No discussion of off-label drug use.
Chapter 8, Hemolytic anemias excluding hemoglobinopathies
Azathioprine, bortezomib, chlorambucil, cyclophosphamide, cyclosporine, danazol, eculizumab, erythropoietin, IV immunoglobulin, mycophenolate mofetil, and rituximab in the treatment of autoimmune hemolytic anemia. Sutimlimab in the treatment of cold agglutinin disease. Eculizumab in the treatment of Shiga toxin–mediated thrombotic microangiopathy (TMA) and drug-induced TMA. Eculizumab in the treatment of drug-induced thrombotic microangiopathy.
Chapter 9, Thrombosis and thrombophilia
No discussion of off-label drug use.
Chapter 10, Bleeding disorders Recombinant factor VIIa for management of bleeding in hemophilia at doses and regimens that are not approved and for other off-label indications, antifibrinolytics for bleeding of unknown cause, and prothrombin complex concentrates for treatment of factor II and X deficiency.
Chapter 11, Disorders of platelet number and function
Desmopressin for inherited platelet function defects and uremic platelets; recombinant factor VIIa for inherited platelet function defects; rituximab for immune thrombocytopenia and thrombotic thrombocytopenic purpura; fondaparinux, bivalirudin, and direct oral anticoagulants for heparin-induced thrombocytopenia.
Chapter 12, Laboratory hematology
No discussion of off-label drug use.
Chapter 13, Transfusion medicine
No discussion of off-label drug use.
Chapter 14, Hematopoietic cell transplantation and cellular therapy
Drug and cellular therapy for hematologic malignancies and hematopoietic cell transplantation.
Chapter 15, Myeloid disorders and inherited bone marrow failure syndromes
Androgens (oxymetholone or danazol) in Fanconi anemia and telomeropathies; granulocyte colony-stimulating factor in non-SCN neutropenia; glucocorticoids in Diamond-Blackfan anemia, autoimmune neutropenias, and histiocytic syndromes; iron chelating agents in congenital dyserythropoietic anemias; interferon-γ in congenital dyserythropoietic anemia type I and Erdheim- Chester disease; plerixafor in WHIM syndrome; intravenous immunoglobin in autoimmune neutropenias; interferon-γ in chronic granulomatous disease; colchicine in familial Mediterranean fever; etoposide, methotrexate, cyclosporine, antithymocyte globulin and dexamethasone in hemophagocytic lymphohistiocytosis; topical steroids, nitrogen mustard, and psoralen in Langerhans cell histiocytosis; vinblastine, methotrexate, and glucocorticoids in Langerhans cell histiocytosis.
Chapter 16, Chronic myeloid leukemia
No discussion of off-label drug use.
Chapter 17, Myeloproliferative neoplasms
Interferon for myeloproliferative neoplasms.
Chapter 18, Acquired marrow failure syndromes: aplastic anemia, paroxysmal nocturnal hemoglobinuria, and myelodysplastic syndromes
Bhumika J. Patel: cyclosporine, rabbit antithymocyte globulin, alemtuzumab, and cyclophosphamide.
Catherine Smith: deferoxamine, deferasirox, deferiprone, epoetin, darbepoetin, filgrastim, tbo-filgrastim, sargramostim, molgramostim, pegfilgrastim, romiplostim, eltrombopag, clofarabine, and venetoclax.
Chapter 19, Acute myeloid leukemia
No discussion of off-label drug use.
Chapter 20, Acute lymphoblastic leukemia and lymphoblastic lymphoma
Blinatumomab, dasatinib, ponatinib, ruxolitinib, bortezomib, venetoclax, navitoclax, and tisagenlecleucel.
Chapter 21, Hodgkin lymphoma
Rituximab for the treatment of lymphocyte-predominant Hodgkin lymphoma; histone deacetylase inhibitors and lenalidomide in relapsed/refractory Hodgkin lymphoma.
Chapter 22, Indolent non-Hodgkin lymphomas
Lenalidomide in follicular lymphoma; rituximab in hairy cell leukemia; bendamustine, brentuximab vedotin, gemcitabine, ibrutinib, and lenalidomide in DLBCL; ibrutinib, and lenalidomide in primary central nervous system lymphoma; alemtuzumab, gemcitabine, lenalidomide, and liposomal doxorubicin in PTCL; mogamulizumab in adult T-cell leukemia/lymphoma.
Chapter 23, Aggressive non-Hodgkin and Burkitt lymphoma Venetoclax in mantle cell lymphoma; mogamolizumab in adult T-cell leukemia/lymphoma; ibrutinib, lenalidomide, mosenutuzumab, glofitamab, and epcoritomab in relapsed/refractory (R/R) diffuse large B-cell lymphoma; and lenalidomide, ibrutinib, pomalidomide, and PD-1 inhibitors in R/R central nervous system lymphoma.
Chapter 24, Chronic lymphocytic leukemia/small lymphocytic lymphoma
Chimeric antigen receptor T cells.
Chapter 25, Plasma cell disorders
Melphalan has no FDA approval for the treatment of myeloma. Venetoclax is not approved for the treatment of myeloma. Proteasome inhibitors are not approved for maintenance therapy for myeloma. The combination of carfilzomib with alkylators is not FDA approved. Isatuximab is not approved in combination with immunomodulatory drugs for myeloma.
Disclosures
As a provider accredited by the Accreditation Council for Continuing Medical Education (ACCME), the American Society of Hematology must ensure balance, independence, objectivity, and scientific rigor in all of the educational activities it sponsors. All individuals in control of content are expected to disclose any financial relationships with ineligible companies that have occurred within the previous 24 months. Ineligible companies are those whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients. If an individual has such an interest, then they must disclose the name of the ineligible company and nature of the relationship (eg, consultant or grantee). An individual who has no such financial relationship with an ineligible company must declare that they have nothing to disclose. The intent of this disclosure is not to prevent an individual with a significant financial or other relationship from making a presentation, but rather to provide readers with information that they can use to make their own judgments. It remains for the audience to determine whether the individual’s interests or relationships may influence the work with regard to exposition or conclusion.
Review disclosure of editor and reviewer financial relationships
Disclosures of author financial relationships are listed below.
Chapter 1, Molecular concepts in hematology and cellular basis of hematopoiesis
Berthold Göttgens: nothing to disclose.
Gabriel Ghiaur: nothing to disclose.
Chapter 2, Consultative hematology 1: hospitalbased and selected outpatient topics
Jennifer R. Green: honoraria: North American ThrombosisForum, Scripps Health. Board membership: EPIC Hematology Steering Board. Grant funding: Health Resources and Services Administration, Centers for Disease Control and Prevention, Patient Centered Outcomes Research Institute, American Thrombosis and Hemostasis Network.
Shannon L. Carpenter: Consultancy: CSL Behring, Kedrion Biopharmaceuticals, HEMA Biologics; Novo Nordisk Pharmaceuticals, Inc; Bayer; Genentech. Membership on board of directors: Hemostasis and Thrombosis Research Society. Grant funding: Shire, CSL Behring.
Chapter 3, Consultative hematology 2: women’s health issues
Leslie Skeith: research funding: CSL Behring. Honoraria: Leo Pharma, Sanofi.
Molly Weidner Mandernach: nothing to disclose.
Andra H. James: research funding: Coagulant Therapeutices. Consulting: Cerus, Octapharma, Tremeau, Hemosonics, Coagulant Therapeutics.
Chapter 4, Hematopoietic growth factors
Sophie Park: research support: Novartis, Pfizer, Takeda, Sandoz, and BMS for research in myelodysplastic syndromes.
Gerald A. Soff: research support: Amgen, Dova/Sobi Pharmaceuticals, and Janssen Scientific Affairs. Advisory boards (in the past 5 years: Amgen, Janssen Scientific Affairs, Bayer Pharmaceuticals, Dova Pharmaceuticals, Bristol Myers Squibb, Pfizer, Novartis, Anthos Therapeutics, and Hengrui (USA) Ltd.
Chapter 5, Iron physiology, iron overload, and the porphyrias
Elizabeta Nemeth: stock ownership: Intrinsic LifeSciences and Silarus Therapeutics. Consultancy: Protagonist Therapeutics, Ionis Pharmaceuticals, Disc Medicine, and Astra- Zeneca-Fibrogen.
Carol Mathew: nothing to disclose.
Chapter 6, Acquired underproduction anemias
Jacquelyn M. Powers: research funding to her institution: American Regent.
Maria Domenica Cappellini: nothing to disclose
Chapter 7, Thalassemia, sickle cell disease, and other hemoglobinopathies
Payal Desai: advisory Board: Pfizer, GBT, Takeda, and Forma. Speaker & Research Project: Novartis.
Rakhi Naik: Rigel, Elsevier.
Jeffrey Lebensburger: consultant: Novartis, Emmaus, Forma Therapeutics, Agios.
Chapter 8, Hemolytic anemias excluding hemoglobinopathies Kevin H. M. Kuo: consultancy: Agios, Alexion, Apellis, Aruvant, Bioverativ/Sanofi, Bluebirdbio, Celgene/BMS, Forma, Novartis, and Pfizer. Member of the scientific advisory board: Agios. Honoraria: Agios, Alexion. and Novartis. Research support: Pfizer. Scientific collaboration: Abfero and Phoenicia Biosciences.
Samuel A. Merrill: member of scientific advisory board: True North Therapeutics, Alexion, and Rigel.
Chapter 9, Thrombosis and thrombophilia
Paul Monagle: nothing to disclose.
Bethany Samuelson Bannow: nothing to disclose.
Chapter 10, Bleeding disorders
Sven Olson: nothing to disclose.
Riten Kumar: consultancy: Bayer.
Chapter 11, Disorders of platelet number and function
Hanny Al-Samkari: consultancy: Agios, Dova, Argenx, Sobi, and Rigel. Research funding: Amgen, Agios, and Dova.
Michele P. Lambert: advisory board membership: Octapharma and Shionogi. Consultancy: Amgen, Novartis, Shionogi, Dova, Principia, Argenx, Rigel, and Bayer. Research funding: Sysmex, Novartis, Rigel, and AstraZeneca.
Chapter 12, Laboratory hematology
Kristi J. Smock: nothing to disclose.
Kenneth D. Friedman: consultancy: Instrumentation Laboratory, Siemens, and Takeda.
Chapter 13, Transfusion medicine
Michelle P. Zeller: medical officer with Canadian Blood Services. Honoraria: Pharmacosmos, Pfizer, and American Society of Hematology; research funding: Canadian Institute of Health Research, Canadian Blood Services, and Pfizer Global Medical Grants.
Stella T. Chou: consultancy: Platelet-Bio. Honoraria: American Society of Hematology, Wolters Kluwer, and Engage Health Media. Research funding: National Institutes of Health and Hyundai Hope on Wheels.
Chapter 14, Hematopoietic cell transplantation and cellular therapy
Irum Khan: honoraria: Takeda and Celgene.
Nirav N. Shah: honoraria and/or travel support: Incyte, Celgene, Lily, and Miltenyi Biotec. Membership on scientific advisory board: Lilly, Kite, Celgene, Legend, Epizyme, Seattle Genetics, and TG Therapeutics. Equity ownership in: Exelixis and Geron. Institutional research support for clinical trials: Miltenyi Biotec.
Matthew Ulrickson: nothing to disclose.
Chapter 15, Myeloid disorders and inherited bone marrow failure syndromes
Boglarka Gyurkocza: clinical trial support: Actinium Pharmaceuticals.
Marcin Wlodarski: nothing to disclose
Chapter 16, Chronic myeloid leukemia
Kendra L. Sweet: research funding: Incyte. Membership on advisory boards: Novartis, Bristol Myers Squibb and Arog. Honorarium: Astellas.
Ehab L. Atallah: research funding: Novartis and Takeda. Consultancy: Pfizer, Celgene, Novartis, and Takeda.
Chapter 17, Myeloproliferative neoplasms
Angela Fleischman: membership on advisory board: PharmaEssentia, CTI, and BMS. Kristen Pettit: membership on an advisory board: PharmaEssentia and Kura Oncology.
Chapter 18, Acquired marrow failure syndromes: aplastic anemia, paroxysmal nocturnal hemoglobinuria, and myelodysplastic syndromes
Bhumika J. Patel: speaker bureau: Alexion and Apellis.
Catherine Smith: research funding: AbbVie. Clinical trial: Celgene.
Chapter 19, Acute myeloid leukemia
Neerav Shukla: nothing to disclose.
Michael F. Walsh: nothing to disclose.
Roland B. Walter: laboratory research grants and/or clinical trial support: Amgen, Aptevo, Celgene, ImmunoGen, Janssen, Jazz, MacroGenics, Pfizer, and Selvita. Ownership interest in: Amphivena. Consultancy: Amphivena, Astellas, Bristol Myers Squibb, Genentech, GlaxoSmithKline, Janssen, Kite, Kronos, and MacroGenics.
Chapter 20, Acute lymphoblastic leukemia and lymphoblastic lymphoma
Oliver G. Ottmann: research funding: Incyte and Amgen.
Liora Schultz: nothing to disclose.
Chapter 21, Hodgkin lymphoma
Celeste Bello: research advisory board (with honorarium): Seattle Genetics.
Pamela B. Allen: research advisory board (with honorarium): Bayer, Daichii Sankyo, Imbrium, and Kyowa Kirin. Research funding: Kyowa Kirin.
Chapter 22, Indolent non-Hodgkin lymphomas
Sairah Ahmed: nothing to disclose.
David T. Yang: consultancy: Novartis.
Chapter 23, Aggressive non-Hodgkin and Burkitt lymphoma
Allison Rosenthal: nothing to disclose.
Amitkumar Mehta: research funding: Incyte, Takeda, Forty Seven Inc/Gilead, Juno Pharmaceuticals/BMS, Celgene/BMS, OncoTartis, Innate Pharmaceuticals, SeaGen, TG Therapeutics, Affimed, Merck, Kite/Gilead, Roche-Genentech, and ADC Therapeutics. Consultancy, speaker, or advisory board member: Gilead, AstraZeneca, Pharmacyclics, SeaGen, Morphosys/Incyte, TG Therapeutics, Kyowa Kirin, and Rigel Pharmaceuticals.
Chapter 24, Chronic lymphocytic leukemia/small lymphocytic lymphoma
Tanya Siddiqi: speaker: Pharmacyclics, AstraZeneca, Bristol Myers Squibb, and Janssen. Advisor: Pharmacyclics, Astra- Zeneca, Bristol Myers Squibb, Juno Therapeutics, Celgene, Kite Pharma, and BeiGene.
Jennifer Woyach: consultant: Pharmacyclics, Janssen, AbbVie, AstraZeneca, BeiGene, Arqule, and Loxo.
Chapter 25, Plasma cell disorders Caitlin Costello: consultancy: BMS, Janssen, Takeda, Karyopharm, and Oncopeptides. Research funding: Celgene, Takeda, Pfizer, and Poseida.
Rafael Fonseca: consultancy: Amgen, BMS, Celgene, Takeda, Bayer, Janssen, Novartis, Pharmacyclics, Sanofi, Karyopharm, Merck, Juno, Kite, Aduro, OncoTracker, Oncopeptides, GSK, and AbbVie. Scientific advisory board member: Adaptive Biotechnologies, Caris Life Sciences, and OncoTracker.
Discussions of off-label drug use are listed below.
The American Society of Hematology requires all authors to disclose any discussion of off-label drug use in their chapters.
Chapter 1, Molecular concepts in hematology and cellular basis of hematopoiesis
No discussion of off-label drug use.
Chapter 2, Consultative hematology 1: hospital based and selected outpatient topics
Jennifer R. Green and Shannon L. Carpenter: corticosteroids and rituximab for use in TTP; ATIII and activated protein C for DIC; cyclophosphamide, corticosteroids, IVIG, and rituximab for CAPS; corticosteroids, intravenous immunoglobulin, rituximab, and thrombopoietin receptor agonists for pediatric ITP; rituximab for adult ITP and TTP; granulocyte-stimulating factor outside of severe congenital neutropenia. Desmopressin for use in platelet function disorders; desmopressin, recombinant FVIIa, prothrombin complex concentrate, activated prothrombin complex concentrate, fibrin glue, ε-aminocaproic acid, and tranexamic acid for use in surgical bleeding and reversal of direct oral anticoagulants; IVIG for posttransfusion purpura; IVIG for drug-induced ITP; conjugated estrogens for use in uremic bleeding; hemoglobin-based oxygen carriers for anemia in patients who refuse blood products.
Chapter 3, Consultative hematology 2: women’s health issues
Almost all medications are considered off-label in pregnancy.
Chapter 4, Hematopoietic growth factors
Sophie Park: deferasirox and lenalidomide in myelodysplastic syndromes.
Gerald A. Soff: all discussion of thrombopoietin-related drugs is currently off-label.
Chapter 5, Iron physiology, iron overload, and the porphyrias
Off-label use of iron chelation therapy.
Chapter 6, Acquired underproduction anemias
No discussion of off-label drug use.
Chapter 7, Thalassemia, sickle cell disease, and other hemoglobinopathies
No discussion of off-label drug use.
Chapter 8, Hemolytic anemias excluding hemoglobinopathies
Azathioprine, bortezomib, chlorambucil, cyclophosphamide, cyclosporine, danazol, eculizumab, erythropoietin, IV immunoglobulin, mycophenolate mofetil, and rituximab in the treatment of autoimmune hemolytic anemia. Sutimlimab in the treatment of cold agglutinin disease. Eculizumab in the treatment of Shiga toxin–mediated thrombotic microangiopathy (TMA) and drug-induced TMA. Eculizumab in the treatment of drug-induced thrombotic microangiopathy.
Chapter 9, Thrombosis and thrombophilia
No discussion of off-label drug use.
Chapter 10, Bleeding disorders Recombinant factor VIIa for management of bleeding in hemophilia at doses and regimens that are not approved and for other off-label indications, antifibrinolytics for bleeding of unknown cause, and prothrombin complex concentrates for treatment of factor II and X deficiency.
Chapter 11, Disorders of platelet number and function
Desmopressin for inherited platelet function defects and uremic platelets; recombinant factor VIIa for inherited platelet function defects; rituximab for immune thrombocytopenia and thrombotic thrombocytopenic purpura; fondaparinux, bivalirudin, and direct oral anticoagulants for heparin-induced thrombocytopenia.
Chapter 12, Laboratory hematology
No discussion of off-label drug use.
Chapter 13, Transfusion medicine
No discussion of off-label drug use.
Chapter 14, Hematopoietic cell transplantation and cellular therapy
Drug and cellular therapy for hematologic malignancies and hematopoietic cell transplantation.
Chapter 15, Myeloid disorders and inherited bone marrow failure syndromes
Androgens (oxymetholone or danazol) in Fanconi anemia and telomeropathies; granulocyte colony-stimulating factor in non-SCN neutropenia; glucocorticoids in Diamond-Blackfan anemia, autoimmune neutropenias, and histiocytic syndromes; iron chelating agents in congenital dyserythropoietic anemias; interferon-γ in congenital dyserythropoietic anemia type I and Erdheim- Chester disease; plerixafor in WHIM syndrome; intravenous immunoglobin in autoimmune neutropenias; interferon-γ in chronic granulomatous disease; colchicine in familial Mediterranean fever; etoposide, methotrexate, cyclosporine, antithymocyte globulin and dexamethasone in hemophagocytic lymphohistiocytosis; topical steroids, nitrogen mustard, and psoralen in Langerhans cell histiocytosis; vinblastine, methotrexate, and glucocorticoids in Langerhans cell histiocytosis.
Chapter 16, Chronic myeloid leukemia
No discussion of off-label drug use.
Chapter 17, Myeloproliferative neoplasms
Interferon for myeloproliferative neoplasms.
Chapter 18, Acquired marrow failure syndromes: aplastic anemia, paroxysmal nocturnal hemoglobinuria, and myelodysplastic syndromes
Bhumika J. Patel: cyclosporine, rabbit antithymocyte globulin, alemtuzumab, and cyclophosphamide.
Catherine Smith: deferoxamine, deferasirox, deferiprone, epoetin, darbepoetin, filgrastim, tbo-filgrastim, sargramostim, molgramostim, pegfilgrastim, romiplostim, eltrombopag, clofarabine, and venetoclax.
Chapter 19, Acute myeloid leukemia
No discussion of off-label drug use.
Chapter 20, Acute lymphoblastic leukemia and lymphoblastic lymphoma
Blinatumomab, dasatinib, ponatinib, ruxolitinib, bortezomib, venetoclax, navitoclax, and tisagenlecleucel.
Chapter 21, Hodgkin lymphoma
Rituximab for the treatment of lymphocyte-predominant Hodgkin lymphoma; histone deacetylase inhibitors and lenalidomide in relapsed/refractory Hodgkin lymphoma.
Chapter 22, Indolent non-Hodgkin lymphomas
Lenalidomide in follicular lymphoma; rituximab in hairy cell leukemia; bendamustine, brentuximab vedotin, gemcitabine, ibrutinib, and lenalidomide in DLBCL; ibrutinib, and lenalidomide in primary central nervous system lymphoma; alemtuzumab, gemcitabine, lenalidomide, and liposomal doxorubicin in PTCL; mogamulizumab in adult T-cell leukemia/lymphoma.
Chapter 23, Aggressive non-Hodgkin and Burkitt lymphoma Venetoclax in mantle cell lymphoma; mogamolizumab in adult T-cell leukemia/lymphoma; ibrutinib, lenalidomide, mosenutuzumab, glofitamab, and epcoritomab in relapsed/refractory (R/R) diffuse large B-cell lymphoma; and lenalidomide, ibrutinib, pomalidomide, and PD-1 inhibitors in R/R central nervous system lymphoma.
Chapter 24, Chronic lymphocytic leukemia/small lymphocytic lymphoma
Chimeric antigen receptor T cells.
Chapter 25, Plasma cell disorders
Melphalan has no FDA approval for the treatment of myeloma. Venetoclax is not approved for the treatment of myeloma. Proteasome inhibitors are not approved for maintenance therapy for myeloma. The combination of carfilzomib with alkylators is not FDA approved. Isatuximab is not approved in combination with immunomodulatory drugs for myeloma.
