Transcript
Page 1: 1.12 添付資料一覧 · 4.3-12 Kalos M and June CH (2013) Adoptive T cell transfer for cancer immunotherapy in the era of synthetic biology. Immunity; 39(1):49-60. 海外 4.3-13

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Page 2: 1.12 添付資料一覧 · 4.3-12 Kalos M and June CH (2013) Adoptive T cell transfer for cancer immunotherapy in the era of synthetic biology. Immunity; 39(1):49-60. 海外 4.3-13

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目 次目 次 .............................................................................................................................2

1 第 3 部 .................................................................................................................................3

2 第 4 部 ...............................................................................................................................15

3 第 5 部 ...............................................................................................................................20

Page 3: 1.12 添付資料一覧 · 4.3-12 Kalos M and June CH (2013) Adoptive T cell transfer for cancer immunotherapy in the era of synthetic biology. Immunity; 39(1):49-60. 海外 4.3-13
Page 4: 1.12 添付資料一覧 · 4.3-12 Kalos M and June CH (2013) Adoptive T cell transfer for cancer immunotherapy in the era of synthetic biology. Immunity; 39(1):49-60. 海外 4.3-13
Page 5: 1.12 添付資料一覧 · 4.3-12 Kalos M and June CH (2013) Adoptive T cell transfer for cancer immunotherapy in the era of synthetic biology. Immunity; 39(1):49-60. 海外 4.3-13
Page 6: 1.12 添付資料一覧 · 4.3-12 Kalos M and June CH (2013) Adoptive T cell transfer for cancer immunotherapy in the era of synthetic biology. Immunity; 39(1):49-60. 海外 4.3-13
Page 7: 1.12 添付資料一覧 · 4.3-12 Kalos M and June CH (2013) Adoptive T cell transfer for cancer immunotherapy in the era of synthetic biology. Immunity; 39(1):49-60. 海外 4.3-13
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添付資料

番号表 題 著者 試験実施期間 試験実施場所

報種類

(国内/

海外)

掲載誌評価/

参考

4.2.2.5 排泄

該当なし - - - - - -

4.2.2.6 薬物動態学的薬物相互作用(非臨床)

該当なし - - - - - -

4.2.2.7 その他の薬物動態試験

該当なし - - - - - -

4.2.3 毒性試験

4.2.3.1 単回投与毒性試験

該当なし - - - - - -

4.2.3.2 反復投与毒性試験

該当なし - - - - - -

4.2.3.3 遺伝毒性試験

該当なし - - - - - -

4.2.3.4 がん原性試験

該当なし - - - - - -

4.2.3.5 生殖発生毒性試験

該当なし - - - - - -

4.2.3.6 局所刺激性試験

該当なし - - - - - -

4.2.3.7 その他の毒性試験

4.2.3.7.1 抗原性試験

該当なし - - - - - -

4.2.3.7.2 免疫毒性試験

該当なし - - - - - -

4.2.3.7.3 毒性発現の機序に関する試験

該当なし - - - - - -

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番号

著者・表 題・掲載誌 報種類

(国内/海外)

4.3 参考文献 (評価/参考の別:参考資料)

4.3-1 Eshhar Z, Waks T, Bendavid A, et al. (2001) Functional expression of chimeric receptor genes in human T cells. J Immunol Methods; 248:67-76. 海外

4.3-2 Fearon D and Carroll M (2000) Regulation of B lymphocyte responses to foreign and self-antigens by the CD19/CD21 complex. Annu Rev Immunol; 18:393-422. 海外

4.3-3 Finney HM, Lawson AD, Bebbington CR, et al. (1998) Chimeric receptors providing both primary and costimulatory signaling in T cells from a single gene product. J Immunol; 161:2791-7.

海外

4.3-4 Finney HM, Akbar AN, Lawson AD (2004) Activation of resting human primary T cells with chimeric receptors: costimulation from CD28, inducible, costimulator, CD134 and CD137 in series with signals from the TCR zeta chain. J Immunol; 172:104-13.

海外

4.3-5 Freedman A, Boyd A, Bieber F, et al. (1987) Normal cellular counterparts of B cell chronic lymphocytic leukemia. Blood; 70(2):418-27. 海外

4.3-6 Friedmann-Morvinski D, Bendavid A, Waks T, et al. (2005) Redirected primary T cells harboring a chimeric receptor require costimulation for their antigen-specific activation. Blood; 105:3087-93.

海外

4.3-7 Gattinoni L, Klebanoff CA, Restifo NP (2012) Paths to stemness: building the ultimate antitumour T cell. Nat Rev Cancer; 12:671–84. 海外

4.3-8 Grupp SA, Kalos M, Barrett D, et al. (2013) Chimeric antigen receptor-modified T cells for acute lymphoid leukemia. N Engl J Med; 368(16):1509-18. 海外

4.3-9 Hombach A, Heuser C, Abken H (2002) The recombinant T cell receptor strategy: insights into structure and function of recombinant immunoreceptors on the way towards an optimal receptor design for cellular immunotherapy. Curr Gene Ther; 2:211-26.

海外

4.3-10 Imai C, Mihara K, Andreansky M, et al. (2004) Chimeric receptors with 4-1BB signaling capacity provoke potent cytotoxicity against acute lymphoblastic leukemia. Leukemia; 18:676-84.

海外

4.3-11 June C, Blazar B, Riley J (2009) Engineering lymphocyte subsets: tools, trials and tribulations. Nat Rev Immunol; 9:704-71. 海外

4.3-12 Kalos M and June CH (2013) Adoptive T cell transfer for cancer immunotherapy in the era of synthetic biology. Immunity; 39(1):49-60. 海外

4.3-13 Kawalekar OU, O’Connor RS, Fraietta JA, et al. (2016) Distinct signaling of coreceptors regulates specific metabolism pathways and impacts memory development in CAR T cells. Immunity; 44:380-90.

海外

4.3-14 Kochenderfer JN, Feldman SA, Zhao Y, et al. (2009) Construction and preclinical evaluation of an anti-CD19 chimeric antigen receptor. J Immunother; 32:689-702. 海外

4.3-15 Krause A, Guo HF, LaTouche JB, et al. (1998) Antigen-dependent CD28 signaling selectively enhances survival and proliferation in genetically modified activated human primary T lymphocytes. J Exp Med; 188(4):619-26.

海外

4.3-16 Ledbetter J, Rabinovitch P, June C, et al. (1988) Antigen-independent regulation of cytoplasmic calcium in B cells with a 12-kDa B-cell growth factor and anti-CD19. Proc Natl Acad Sci USA; 85:1897-901.

海外

4.3-17 Long AH, Haso WM, Shern JF, et al. (2015) 4-1BB costimulation ameliorates T cell exhaustion induced by tonic signaling of chimeric antigen receptors. Nature Med; 21(6)581-93.

海外

4.3-18 Maher J, Brentjens RJ, Gunset G, et al. (2002) Human T-lymphocyte cytotoxicity and proliferation directed by a single chimeric TCRz/CD28 receptor. NatBiotech.; 20:70-5. 海外

4.3-19 Maude SL, Frey N, Shaw PA (2014) Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia. N Engl J Med.; 371(16)1507-17. 海外

4.3-20 Milone MC, Fish JD, Carpenito C, et al. (2009) Chimeric receptors containing CD137 signal transduction domains mediate enhanced survival of T cells and increased antileukemic efficacy in vivo. Mol Ther; 17(8):1453-64.

海外

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4.3-21 Nicholson I, Lenton K, Little D, et al. (1997) Construction and characterisation of a functional CD19 specific single chain Fv fragment for immunotherapy of B lineage leukemia and lymphoma. Mol Immunol; 34(16-17):1157-65.

海外

4.3-22 Porter D, Levine B, Kalos M, et al. (2011) Chimeric antigen receptor-modified T cells in chronic lymphoid leukemia. N Engl J Med; 365(8):725-33. 海外

4.3-23 Porter D, Hwang WT, Frey NV, et al. (2015) Chimeric antigen receptor T cells persist and induce sustained remissions in relapsed refractory chronic lymphocytic leukemia. Sci Transl Med;7(303):303ra139.

海外

4.3-24 Pule M, Finney H, Lawson A (2003) Artificial T-cell receptors. Cytotherapy; 5(3):211-26. 海外

4.3-25 Pulle G, Vidric M, Watts TH (2006) IL-15-dependent induction of 4-1BB promotes antigen-independent CD8 memory T cell survival. J Immunol; 176(5):2739-48. 海外

4.3-26 Sadelain M, Riviere I, Brentjens R (2003) Targeting tumors with genetically enhanced T lymphocytes. Nat Rev Cancer; 3:35-45. 海外

4.3-27 Schuster SJ, Svoboda J, Nasta S, et al. (2015) Phase IIa trial of chimeric antigen receptor modified T cells directed against CD19 (CTL019) in patients with relapsed or refractory CD19+ lymphomas. J Clin Oncol; 33(15),suppl 8516.

海外

4.3-28 Shuford WW, Klussman K, Tritchler DD, et al. (1997) 4-1BB costimulatory signals preferentially induce CD8(+) T cell proliferation and lead to the amplification in vivo of cytotoxic T cell responses. J Exp Med; 186(1):47-55.

海外

4.3-29 Stamenkovic I and Seed B (1988) CD19, the earliest differentiation antigen of the B cell lineage, bears three extracellular immunoglobulin-like domains and an epstein-barr virus-related cytoplasmic tail. J Exp Med; 168:1205-10.

海外

4.3-30 Teachey D, Obzut D, Cooperman J, et al. (2006) The mTOR inhibitor CCI-779 induces apoptosis and inhibits growth in preclinical models of primary adult human ALL. Blood; 107(3):1149-55.

海外

4.3-31 Uckun FM, Jaszcz W, Ambrus JL, et al. (1988) Detailed studies on expression and function of CD19 surface determinant by using B43 monoclonal antibody and the clinical potential of anti-CD19 immunotoxins. Blood; 71(1):13-29.

海外

4.3-32 Geginat J, Paroni M, Maglie S, et al. (2014) Plasticity of human CD4 T cell subsets. Front Immunol; 5:630. 海外

4.3-33 Katz BZ and Herishanu Y (2014) Therapeutic targeting of CD19 in hematological malignancies: past,present, future and beyond. Leuk Lymphom. 55(5):999-1006.

海外

4.3-34 Wang K, Wei G, Liu D (2012) CD19: a biomarker for B cell development, lymphoma diagnosis andtherapy. Exp Hematol Oncol. 1(1):36.

海外

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5.4 参考文献 (評価/参考の別:参考資料)

5.4-1 Anderlini P, Rizzo JD, Nugent ML, et al. (2001) Peripheral blood stem cell donation: an analysis from the International Bone Marrow Transplant Registry (IBMTR) and European Group for Blood and Marrow Transplant (EBMT) databases. Bone Marrow Transplant; 27(7):689-92.

海外

5.4-2 Appelbaum FR, Rosenblum D, Arceci RJ, et al. (2007) End points to establish the efficacy of new agents in the treatment of acute leukemia. Blood; 109(5):1810-6. 海外

5.4-3 Barrington SF, Mikhaeel NG, Kostakoglu L, et al. (2014) Role of imaging in the staging and response assessment of lymphoma: consensus of the International Conference on Malignant Lymphomas Imaging Working Group. J Clin Oncol; 32(27):3048-58.

海外

5.4-4 Bhojwani D, Sposto R, Shah N, et al. (2017) Inotuzumab ozogamicin in pediatric patients with relapsed/refractory acute lymphoblastic leukemia (R/R ALL) [abstract]. J Clin Oncol; 35:15_suppl, 10512.

海外

5.4-5 Blennow O, Ljungman P, Sparrelid E, et al. (2014) Incidence, risk factors, and outcome of bloodstream infections during the pre-engraftment phase in 521 allogeneic hematopoietic stem cell transplantations. Transpl Infect Dis; 16(1):106-14.

海外

5.4-6 Bondarenko SN, Moiseev IS, Slesarchuk OA, et al. (2016) Allogeneic hematopoietic stem cell transplantation in children and adults with acute lymphoblastic leukemia. Cellular Therapy and Transplantation; 5(2):12-20.

海外

5.4-7 Brookmeyer R and Crowley JJ (1982) A confidence interval for the median survival time. Biometrics; 38:29-41. 海外

5.4-8 Brudno JN and Kochenderfer JN (2016) Toxicities of chimeric antigen receptor T cells: recognition and management. Blood; 127(26):3321-30. 海外

5.4-9 Cheson BD, Bennett JM, Kopecky KJ, et al. (2003) Revised recommendations of the International Working Group for diagnosis, standardization of response criteria, treatment outcomes, and reporting standards for therapeutic trials in acute myeloid leukemia. J Clin Oncol; 21(24):4642-9.

海外

5.4-10 Cheson BD, Fisher RI, Barrington SF, et al. (2014) Recommendations for initial evaluation, staging, and response assessment of Hodgkin and Non-Hodgkin lymphoma: The Lugano classification. J Clin Oncol; 32(27):3059-67.

海外

5.4-11 Chessells JM, Veys P, Kempski H, et al. (2003) Long-term follow-up of relapsed childhood acute lymphoblastic leukaemia. Br J Haematol; 123(3):396-405. 海外

5.4-12 clin-spa-2202-20140723 Letter of re-concurrence of Study B2202 SPA (Jul 2014) 海外

5.4-13 Coiffier B and Sarkozy C (2016) Diffuse large B-cell lymphoma: R-CHOP failure—what to do? Hematology Am Soc Hematol Educ Program; 2016(1):366-78. 海外

5.4-14 Correspond-FDA-pre-sBLA-20170804 Minutes of the pre-sBLA meeting with the FDA (Aug 2017) 海外

5.4-15 Correspond-FDA-pre-Bla-20161121 Minutes of the pre-BLA meeting with the FDA (Nov 2016) 海外

5.4-16 Correspond-FDA-Type-B-20160502 Minutes of the Type B meeting with the FDA (May 2016) 海外

5.4-17 Crotta A, Keir C, Cleret J, et al. (2017) Disease Characteristics and Overall Survival in Pediatric Patients with Relapsed and Refractory B-Cell Acute Lymphoblastic Leukemia after Stem Cell Transplantation [abstract]. Biol Blood Marrow Transplant; 23:S18-S391.

海外

5.4-18 Crump M, Neelapu SS, Farroq U, et al. (2017) Outcomes in refractory diffuse large B-cell lymphoma: Results from the international SCHOLAR-1 study. Blood; 130(16):1800-8.

海外

5.4-19 Davila ML, Riviere I, Wang X, et al. (2014) Efficacy and toxicity management of 19–28z CAR T cell therapy in B cell acute lymphoblastic leukemia. Sci Transl Med; 6(224):224ra25.

海外

5.4-20 Eapen M, Giralt SA, Horowitz MM, et al. (2004) Second transplant for acute and chronic leukemia relapsing after first HLA-identical sibling transplant. Bone Marrow Transplant; 34(8):721-7.

海外

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5.4-21 EMA SA 2014 Minutes of the Scientific Advice meeting with the CHMP (Apr 2014) 海外

5.4-22 EMA SA 2017 Minutes of the pre-submission meeting with the EMA (Sep 2017) 海外

5.4-23 Fenske TS, Ahn KW, Graff TM, et al. (2016) Allogenic transplantation provides durable remission in a subset of DLBCL patients relapsing after autologous transplantation. Br J Haematol; 174(2):235-48.

海外

5.4-24 Fitzgerald JC, Weiss, SL, Maude SL, et al. (2017) Cytokine release syndrome after chimeric antigen receptor T cell therapy for acute lymphoblastic leukemia. Crit Care Med;45(2):e124-e131.

海外

5.4-25 Frey N, Grange S, Woodworth T (2010) Population pharmacokinetic analysis of tocilizumab in patients with rheumatoid arthritis. J Clin Pharmacol; 50:754-66. 海外

5.4-26 Friedberg JW (2011) Relapsed/refractory diffuse large B-cell lymphoma. Hematology AM Soc Hematol Educ Program. 2011:498-505. 海外

5.4-27 Ghielmini M, Vitolo U, Kimby E, et al. (2013) ESMO guidelines consensus conference on malignant lymphoma 2011 part 1: diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL) and chronic lymphocytic leukemia (CLL). Ann Oncol; 24(3):561-76.

海外

5.4-28 Gill S and June CH (2015) Going viral: chimeric antigen receptor T-cell therapy for hematological malignancies. Immunol Rev; 263(1):68-89. 海外

5.4-29 Gisselbrecht C, Glass B, Mounier N, et al. (2010) Salvage regimens with autologous transplantation for relapsed large B-cell lymphoma in the rituximab era. J Clin Oncol; 28(27):4184-90.

海外

5.4-30 Harrison CN, Mesa RA, Kiladjian JJ, et al. (2013) Health-related quality of life andsymptoms in patients with myelofibrosis treated with ruxolitinib versus best available therapy. Br J Haematol; 162(2):229-39.

海外

5.4-31 Hijiya N, Thomson B, Isakoff MS, et al. (2011) Phase 2 trial of clofarabine in combination with etoposide and cyclophosphamide in pediatric patients with refractory or relapsed acute lymphoblastic leukemia. Blood; 118(23):6043-9.

海外

5.4-32 Horibe K, Saito AM, Takimoto T, et al. (2013) Incidence and survival rates of hematological malignancies in Japanese children and adolescents (2006-2010): based on registry data from the Japanese Society of Pediatric Hematology. Int J Hematol; 98(1):74-88.

海外

5.4-33 Hunger SP and Mullighan CG (2015) Acute lymphoblastic leukemia in children. N Engl J Med; 373(16):1541-52. 海外

5.4-34 Inagaki J, Fukano R, Noguchi M, et al. (2015) Hematopoietic stem cell transplantation following unsuccessful salvage treatment for relapsed acute lymphoblastic leukemia in children. Pediatr Blood Cancer; 62(4):674-9.

海外

5.4-35 Jeha S, Gaynon PS, Razzouk BI, et al. (2006) Phase II study of clofarabine in pediatric patients with refractory or relapsed acute lymphoblastic leukemia. J Clin Oncol; 24(12):1917-23.

海外

5.4-36 Ko RH, Ji L, Barnette P, et al. (2010) Outcome of patients treated for relapsed or refractory acute lymphoblastic leukemia: a Therapeutic Advances in Childhood Leukemia Consortium study. J Clin Oncol; 28(4):648-54.

海外

5.4-37 Lan KK and DeMets DL (1983) Discrete sequential boundaries for clinical trials. Biometrika; 70(3):659-63. 海外

5.4-38 Lee DW, Gardner R, Porter DL, et al. (2014) Current concepts in the diagnosis and management of cytokine release syndrome. Blood; 124(2):188-95. 海外

5.4-39 Locatelli F, Testi AM, Bernardo ME, et al. (2009) Clofarabine, cyclophosphamide and etoposide as single-course re-induction therapy for children with refractory/multiple relapsed acute lymphoblastic leukaemia. Br J Haematol; 147(3):371-8.

海外

5.4-40 Lymphoma Study Group of Japanese Pathologists (2000) The world health organization classification of malignant lymphomas in japan: incidence of recently recognized entities. Pathol Int; 50(9):696-702.

海外

5.4-41 Martin A, Morgan E, Hijiya N (2012) Relapsed or refractory pediatric acute lymphoblastic leukemia: current and emerging treatments. Pediatr Drugs; 14(6):377-87. 海外

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5.4-42 Maude SL, Teachey DT, Porter DL, et al. (2015) CD19-targeted chimeric antigen receptor T-cell therapy for acute lymphoblastic leukemia. Blood; 125(26):4017-23. 海外

5.4-43 Meeske K, Katz ER, Palmer SN, et al. (2004) Parent proxy-reported health-related quality of life and fatigue in pediatric patients diagnosed with brain tumors and acute lymphoblastic leukemia. Cancer; 101(9):2116-25.

海外

5.4-44 MHRA NSA 2017 Minutes of the pre-submission meeting with the Co-Rapporteur (MHRA) (Sep 2017) 海外

5.4-45 Miano M, Pistorio A, Putti MC, et al. (2012) Clofarabine, cyclophosphamide and etoposide for the treatment of relapsed or resistant acute leukemia in pediatric patients. Leuk Lymphoma; 53(9):1693-8.

海外

5.4-46 NoMA NSA 2017 Minutes of the pre-submission meeting with the Rapporteur (NoMA) (Sep 2017) 海外

5.4-47 Peeters M and Poon A (1987) Down syndrome and leukemia: unusual clinical aspects and unexpected methotrexate sensitivity. Eur J Pediatr; 146(4):416–22. 海外

5.4-48 PEI NSA 2013 Minutes of the Scientific Advice meeting with the Paul-Ehrlich Institute (Oct 2013) 海外

5.4-49 Pfreundschuh M, Kuhnt E, Trümper L, et al. (2011) CHOP-like chemotherapy with or without rituximab in young patients with good-prognosis diffuse large-B-cell lymphoma: 6-year results of an open-label randomised study of the MabThera International Trial (MInT) Group. Lancet Oncol; 12(11):1013-22.

海外

5.4-50 Pickard AS, Neary MP, Cella D (2007) Estimation of minimally important differences in EQ-5D utility and VAS scores in cancer. Health Qual Life Outcomes; 5:70. 海外

5.4-51 Pui CH, Robison LL, Look AT (2008) Acute lymphoblastic leukemia. Lancet; 371(9617):1030-43. 海外

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