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Review

Acute progranulocytic leukaemia: a model for molecular medicine

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Pages 135-150 | Published online: 25 Feb 2005

Bibliography

  • GRIGANI F, FAGIOLI M, ALCALY M et al.: Acute promye-locytic leukaemia: from genetics to treatment. Blood (1994) 83:10–25.
  • •An outstanding review integrating the molecular, biological and clinical features of APL with the clinical outcome from molecularly-targeted therapy (namely, ATRA).
  • WARRELL RJ, DE THE H, WANG Z, DEGOS L: Acute pro-myelocytic leukaemia. New Engl. J. Med (1993) 329:177–189.
  • ROWLEY J, GOLOMB H, DOUGHERTY C: 15;17 transloca-tion, a consistent chromosomal change in acute pro-myelocytic leukaemia. Lancet (1977) 1:549–550.
  • DE THE H, CHOMIENNE C, LANOTTE M, DEGOS L, DE-JEAN A: The 015;17) translocation of acute promyelo-cytic leukaemia fuses the retinoic acid receptor-a gene to a novel transcribed locus. Nature (1990) 347:558–562.
  • ••Original study identifying the molecular nature of t(15;17), fusing the RARA and PML genes.
  • GODDARD A, BORROW J, FREEMONT P, SOLOMON E: Characterization of a zinc finger gene disrupted by the 015;17) in acute promyelocytic leukaemia. Science (1991) 254:1371–1374.
  • BIONDI A, RAMBALDI A, ALCALAY M et al.: RAR-a gene rearrangements as a genetic marker for diagnosis and monitoring in acute promyelocytic leukaemia. Blood (1991) 77:1418–1422.
  • CHEN S, ZHU Y, TONG J et al.: Rearrangements in the second intron of the RARA gene are present in a large majority of patients with acute promyelocytic leukae-mia and are used as molecular marker for retinoic acid-induced leukemic cell differentiation. Blood (1991) 78:2696–2701.
  • DIVERIO D, LO COCO F, D'ADAMO F et al.: Identificationof DNA rearrangements at the retinoic acid receptor-a (RAR-a) locus in all patients with acute promyelocytic leukaemia (APL) and mapping of APL breakpoints within the RAR-a second intron. Blood (1992) 79:3331–3336.
  • CASTAIGNE S, BALITRAND N, DE THE H et al.: A PML/reti-noic acid receptor-a fusion transcript is constantly de-tected by RNA-based polymerase chain reaction in acute promyelocytic leukaemia. Blood (1992) 79:3110–3115.
  • CHANG K, LU J, WANG G et al.: The 015;17) breakpoint in acute promyelocytic leukaemia cluster within two different sites of the myl gene: targets for the detection of minimal residual disease by polymerase chain reac-tion. Blood (1992) 79:554–558.
  • HUANG W, SUN G, LI X et al.: Acute promyelocytic leu-kaemia: clinical relevance of two major PML/RARa iso-forms and detection of minimal residual disease by retrotranscriptase/polymerase chain reaction to pre-dict relapse. Blood (1993) 82:1264–1269.
  • GALLAGHER R, WILLMAN C, SLACK J et al: Association of PML-RARA fusion mRNA type with pretreatment he-matologic characteristics but not treatment outcome in acute promyelocytic leukaemia: an intergroup mo-lecular study. Blood (1997) 90:1656–1663.
  • SLACK J, ARTHUR D, LAWRENCE D et al.: Secondary cyto-genetic changes in acute promyelocytic leukaemia-prognostic importance in patients treated with che-motherapy alone and association with the intron 3 breakpoint of the PML gene: a Cancer and Leukaemia Group B study. J. Clin. Oncol. (1997) 15:1786–1795.
  • SLACK J, YU M: Constitutive expression of the promye-locytic leukaemia-associated oncogene PML-RARA in TF-1 cells: isoform-specific and retinoic acid-dependent effects on growth; bc1-2 expression, and apoptosis. Blood (1998) 91:3347–3356.
  • GRIGANI F, FERRUCCI P, TESTA U et al.: The acute pro-myelocytic leukaemia specific PML/RARa fusion pro-tein inhibits differentiation and promotes survival of myeloid precursor cells. Cell (1993) 74:423–431.
  • WANG Z, DELVA L, GABOLI M et al.: Role of PML in cell growth and the retinoic acid pathway. Science (1998) 279:1547–1551.
  • GRISOLANO J, WESSELSCHMIDTR, PELICCI P, LEY T: Al-tered myeloid development and acute leukaemia in transgenic mice expressing PML-RARA under control of cathepsin G regulatory sequences. Blood (1997) 89:376–387.
  • DANIEL M, KOKEN M, TOMAGNE O et al.: PML protein expression in hematopoietic and acute promyelocytic leukaemia cells. Blood (1993) 82:1858–1867.
  • CHANG K, FAN Y, ANDREEFF M, LIU J, MU Z: The PML gene encodes a phosphoprotein associated with the nuclear matrix. Blood (1995) 85:3646–3653.
  • DYCK J, WARRELL RJ, EVANS R, MILLER WJ: Rapid diagno-sis of acute promyelocytic leukaemia by immunohis-tochemical localization of PML/RARA protein. Blood (1995) 86:862–867.
  • ALCALY M, TOMASSINI L, COLOMBO E et al.: The pro-myelocytic leukaemia gene product (PML) forms sta-ble complexes with the retinoblastoma protein. Mol. Cell Biol. (1998) 18:1084–1093.
  • CHEN S, ZELENT A, TONG J et al.: Rearrangements of the retinoic acid receptor alpha and promyelocytic leu-kaemia zinc finger genes resulting from t(11;17)(q23;q21) in a patient with acute promyelo-cytic leukaemia. J. Clin. Invest. (1993) 91:2260–2267.
  • LICHT J, CHOMIENNE C, GOY A et al.: Clinical and mo-lecular characterization of a rare syndrome of acute promyelocytic leukaemia associated with transloca-tion (11;17). Blood (1995) 85:1083–1094.
  • REDNER R, RUSH E, FAAS S, RUDERT W, COREY S: The05;17) variant of acute promyelocytic leukemia ex-presses a nucleophosmin-retinoic acid receptor fu-sion. Blood (1996) 87:882–886.
  • WELLS R, CATZAVELOS C, KAMEL-REID S: Fusion of reti-noic acid receptor to NuMA, the mitotic apparatus protein, by a variant translocation in acute promyelo-cytic leukaemia. Nature Genet. (1997) 17:109–113.
  • KARP J, SMITH M: The molecular pathogenesis of treatment-induced (secondary) leukaemias: founda-tions for treatment and prevention. Semin. Oncol. (1997) 24:103–113.
  • GILL-SUPER H, MCCABE N, THIRMAN M et al.: Rearrange-ment of the MLL gene in acute myeloid leukaemia in patients previously treated with agents targeting DNA-topoisomerase II. Blood (1993) 82:3705–3711.
  • SHAKNOVICH R, YEYATI P, WINS S et al.: The promyelo-cytic leukaemia zinc finger protein affects myeloid cell growth, differentiation, and apoptosis. Mol. Cell Biol. (1998) 18:5533–5545.
  • TENEN D, HROMAS R, LICHT J, ZHANG D: Transcription factors, normal myeloid development, and leukaemia. Blood (1997) 90:489–519.
  • KOKEN M, REID A, QUIGNON F et al.: Leukaemia associ-ated retinoic acid receptor a fusion partners, PML and PLZF, heterodimerize and colocalize to nuclear bodies. Proc. Natl. Acad. Sci. (1997) 94:10255–10260.
  • GUIDEZ F, HUANG W, TONG J et al.: Poor response to all-trans retinoic acid in a 011;17) PLZF/RARa patient. Leukaemia (1994) 8:312–317.
  • GUIDEZ F, IVINS S, ZHU J et al.: Reduced retinoic acid sensitivities of nuclear receptor corepressor binding to PML-and PLZF-RARA underlie molecular pathogene-sis and treatment of acute promyelocytic leukaemia. Blood (1998) 91:2634–2642.
  • HILLESTAD L: Acute Promyelocytic Leukaemia. Acta. Medica. Scandinavica (1957) 159:189–194.
  • •Classical paper describing APL as a distinct clinical entity and defining the unique APL-associated haemorrhagic diathesis.
  • DALY PA, SCHIFFER CA, WIERNIK PH: Acute promyelo-cytic leukaemia-clinical management of 15 patients. Am. J. Hematol. (1980) 8:347–359.
  • CORDONNIER C, VERNANT JP, BRUN B et al.: Acute pro-myelocytic leukaemia in 57 previously untreated pa-tients. Cancer (1985) 55:18–25.
  • CUNNINGHAM I, GEE TS, REICH LM et al.: Acute promye-locytic leukaemia: treatment results during a decade at Memorial Hospital. Blood (1989) 73:1116–1122.
  • KANTARJIAN HM, KEATING MJ, WALTERS RS et al.: Acute promyelocytic leukaemia. M.D. Anderson Hospital ex-perience. Am. J. Med. (1986) 80:789–797.
  • STONE RM, MAGUIRE M, GOLDBERG MA et al: Complete remission in acute promyelocytic leukaemia despite persistence of abnormal bone marrow promyelocytes during induction therapy: experience in 34 patients. Blood (1988) 71:690–696.
  • RODEGHIERO F, AVVISATI G, CASTAMAN G, BARBUI T, MANDELLI F: Early deaths and anti-hemorrhagic treat-ments in acute promyelocytic leukaemia. A GIMEMA retrospective study in 268 consecutive patients. Blood (1990) 75:2112–2117.
  • AVVISATI G, MANDELLI F, PETTI MC et al.: Idarubicin (4-demethoxydaunorubicin) as single agent for remis-sion induction of previously untreated acute promye-locytic leukaemia: a pilot study of the Italian co-operative group GIMEMA. Eur. J. Haematol. (1990) 44:257–260.
  • WILDE JT, DAVIES JM: Haemostatic problems in acute leukaemia. Blood Rev. (1990) 4:245–51.
  • TALLMAN MS: Deciphering the pathogenesis of coagu-lation dysfunction in leukaemia. Leuk. Res. (1996) 20:13–16.
  • BERNARD J, WEIL M, BOIRON M et al.: Acute promyelo-cytic leukaemia: results of treatment by daunorubicin. Blood (1973) 41:489–496.
  • ••First demonstration that APL is highly sensitive to daunoru-bicin, and that a high remission rate and long remission du-rations can be achieved with daunorubicin therapy, plus recognition and management of DIC.
  • SANZ MA, JARQUE I, MARTIN G et al.: Acute promyelo-cytic leukaemia. Therapy results and prognostic fac-tors. Cancer (1988) 61:7–13.
  • KANTARJIAN HM, KEATING MJ, MCCREDIE KB et al.: A characteristic pattern of leukemic cell differentiation without cytoreduction during remission induction in acute promyelocytic leukaemia. J. Clin. Oncol (1985) 3:793–798.
  • DAENEN S, VELLENGA E, VAN DOBBENBURGH OA, HALIE MR: Retinoic acid as antileukemic therapy in a patient with acute promyelocytic leukaemia and As-pergillus pneumonia. Blood (1986) 67:559–61.
  • FLYNN PJ, MILLER WJ, WEISDORF DJ et al.: Retinoic acid treatment of acute promyelocytic leukaemia: in vitro and in vivo observations. Blood (1983) 62:1211–1217.
  • FONTANA JA, ROGERS JSD, DURHAM JP: The role of 13 cis-retinoic acid in the remission induction of a patient with acute promyelocytic leukaemia. Cancer (1986) 57:209–17.
  • HUANG ME, YE YC, CHEN SR et al.: Use of all-trans reti-noic acid in the treatment of acute promyelocytic leu-kaemia. Blood (1988) 72:567–572.
  • ••Original report of the clinical efficacy of ATRA in inducingclinically and haematologically complete remissions in APL.
  • WANG ZY, SUN GL, LU JX et al.: Treatment of acute pro-myelocytic leukaemia with all-trans retinoic acid in China. Nouv. Rev. Fr. Hematol (1990) 32:34–36.
  • CASTAIGNE S, CHOMIENNE C, DANIEL MT et al.: All-trans retinoic acid as a differentiation therapy for acute pro-myelocytic leukaemia. I. Clinical results. Blood (1990) 76:1704–1709.
  • •Detailed description of clinical differentiation with ATRA in-duction therapy for APL.
  • CHOMIENNE C, BALLERINI P, BALITRAND N et al.: All-trans-retinoic acid as a differentiation therapy for acute promyelocytic leukaemia. II In vitro studies: structure-function relationship. Blood (1990) 76:1710-1717.
  • •In vitro laboratory correlates accompanying clinical trial of ATRA [51].
  • DEGOS L, DOMBRET H, CHOMIENNE C et al.: All-trans re-tinoic acid as a differentiating agent in the treatment of acute promyelocytic leukaemia. Blood (1995) 85:2643–2653.
  • RAELSON J, NERVI C, ROSENAUER A et al.: The PML/RARA oncoprotein is a direct molecular target of retinoic acid in acute promyelocytic leukaemia. Blood (1996) 88:2826–2832.
  • NERVI C, FEVVARA F, FANELLI M et al.: Caspases mediate retinoic acid-induced degradation of the acute pro-myelocytic leukaemia PML/RARA fusion protein. Blood (1998) 92:2244–2251.
  • BENEDETTI L, GRIGNANI F, SCICCHITANO B et al.:Retinoid-induced differentiation of acute promyelo-cytic leukaemia involves PML-RARA-mediated in-crease of Type II transglutaminase. Blood (1996) 87:1939–1950.
  • VYAS R, FRANKEL S, AGBOR P et al.: Probing the pathobi-ology of response to all-trans retinoic acid in acute promyelocytic leukaemia: premature chromatin con-densation/fluorescence in situ hybridization analysis. Blood (1996) 87:218–226.
  • FENAUX P, LE DELEY MC, CASTAIGNE S et al.: Effect of alltrans retinoic acid in newly diagnosed acute promye-locytic leukaemia. Results of a multicenter random-ized trial. European APL 91 Group. Blood (1993) 82:3241–3249.
  • TALLMAN MS, ANDERSEN JW, SCHIFFER CA et al.: All-trans-retinoic acid in acute promyelocytic leukaemia. New Engl. J. Med. (1997) 337:1021–1028.
  • ••Large clinical trial defining the critical role of ATRA in theachievement and duration of complete remission in APL.
  • BIONDI A, RAMBALDI A, PANDOLFI P et al.: Molecular monitoring of the myl/retinoic acid receptor-a fusion gene in acute promyelocytic leukaemia by polymerase chain reaction. Blood (1992) 80:492–497.
  • DIVERIO D, ROSSI V, AVVISATI G et al.: Early detection of relapse by prospective reverse transcriptase-polymerase chain reaction analysis of the PML/RARa fusion gene in patients with acute promyelocytic leu-kaemia enrolled in the GlMEMA-AIEOP multicenter 'AIDS' trial. Blood (1998) 92:784–789.
  • MILLER WJ, LEVINE K, DEBLASIO A et al.: Detection ofminimal residual disease in acute promyelocytic leu-kaemia by a reverse transcriptase polymerase chain reaction assay for the PML/RARA fusion mRNA. Blood (1993) 82:1689–1694.
  • SMITH M, PARKINSON D, CHESON B, FRIEDMAN M: Reti-noids in cancer therapy. J. Clin. Oncoi (1992) 10:839–845.
  • DELVA L, CORNIC M, BALITRAND N et al. Resistance to all-trans retinoic acid (ATRA) therapy in relapsing acute promyelocytic leukemia: study of in vitro ATRA sensitivity and cellular retinoic acid binding protein levels in leukemic cells. Blood (1993) 82:2175–2181.
  • ROSENAUER A, RAELSON J, NERVI C et al.: Alterations inexpression, binding to ligand and DNA, and transcrip-tional activity of rearranged and wild-type retinoid re-ceptors in retinoid-resistant acute promyelocytic leukaemia cell lines. Blood (1996) 88:2671–2682.
  • SHAO W, BENEDETTI L, LAMPH W, NERVI C, MILLER WJ: Aretinoid-resistant acute promyelocytic leukaemia sub-clone expresses a dominant negative PML-RARa muta-tion. Blood (1997) 89:4282–4289.
  • DING W, LI Y, NOBILE L et al.: Leukaemic cellular reti-noic acid resistance and missense mutations in the PML-RARA fusion gene after relapse of acute promye-locytic leukaemia from treatment with all-trans-retinoic acid and intensive chemotherapy. Blood (1998) 92:1172–1183.
  • IMAIZUMI M, SUZUKI H, YOSHINARI M et al.: Mutationsin the E-domain of RARA portion of the PML/RARA chi-meric gene may confer clinical resistance to all-trans-retinoic acid in acute promyelocytic leukaemia. Blood (1998) 92:374–382.
  • CHEN G, SHI X, TANG W et al.: Use of arsenic trioxide(As203) in the treatment of acute promyelocytic leu-kaemia (APL): I As203 exerts dose-dependent dual ef-fects on APL cells. Blood (1997) 89:3345–3355.
  • CHEN G, ZHU J, SHI X et al.: In vitro studies on cellularand molecular mechanisms of arsenic trioxide (As203) in the treatment of acute promyelocytic leu-kaemia: As203 induces NB4 cell apoptosis with down-regulation of bc1-2 expression and modulation of PML-RARa/PML proteins. Blood (1996) 88:1052–1061.
  • GIANNI M, KOKEN M, CHELBI-ALIX M et al.: Combinedarsenic and retinoic acid treatment enhances differen-tiation and apoptosis in arsenic-resistant NB-4 cells. Blood (1998) 91:4300–4310.
  • SHAO W, FANELLI M, FERRARA F et al.: Arsenic trioxideas an inducer of apoptosis and loss of PML/RARa pro-tein in acute promyelocytic leukaemia cells. J. Nati Cancer Inst. (1998) 90:124–133.
  • SHEN Z, CHEN G, NI J et al.: Use of arsenic trioxide(As203) in the treatment of acute promyelocytic leu-kaemia (APL): II Clinical efficacy and pharmacokinet-ics in relapsed patients. Blood (1997) 80:3354–3360.
  • WANG Z, RIVI R, DELVA L et al.: Arsenic trioxide and me-larsoprol induce programmed cell death in myeloid leukaemia cell lines and function in a PML and PML-RARA independent manner. Blood (1998) 92:1497–1504.
  • DAI J, WEINBERG RS, WAXMAN S, JING Y: Malignant cellscan be sensitized to undergo growth inhibition and apoptosis by arsenic trioxide through modulation of the glutathione redox system. Blood (1999) 93:268–277.
  • MULLER S, MILLER WH JR, DEJEAN A: Trivalent antimoni-als induce degradation of the PML-RARa oncoprotein and reorganization of the promyelocytic leukaemia nuclear bodies in acute promyelocytic leukaemia. Blood (1998) 92:4308–4316.
  • MAITKAINEN S, RONNI T, HURME M, PINE R, JULKUNEN I: Retinoic acid activates interferon regulatory factor-1 gene expression in myeloid cells. Blood (1996) 88:114–123.
  • NASON-BURCHENAL K, GANDINI D, BOTTO D et al.: In-terferon augments PML and PMURARa expression in normal myeloid and acute promyelocytic cells and co-operates with all-trans retinoic acid to induce matura-tion of a retinoid-resistant promyelocytic cell line. Blood (1996) 88:3926–3939.
  • CHIN Y, KITAGAWA M, SU W-C et al.: Cell growth arrest and induction of cyclin-dependent kinase inhibitor p21WAF-1/C1P-1 mediated by STAT-1. Science (1996) 272:719–722.
  • GIANNI M, TERAO M, FORTINA I et al.: Statl is induced and activated by all-trans retinoic acid in acute pro-myelocytic leukaemia cells. Blood (1997) 89:1001–1012.
  • GRIGNANI F, DE MATTELS S, NERVI C et al: Fusion pro-teins of the retinoic acid receptor-a recruit histone deacetylase in promyelocytic leukaemia. Nature (1998) 391:815–818.
  • LIN R, NAGY L, INOUE S et al.: Role of histone deacety-lase complex in acute promyelocytic leukaemia. Na-ture (1998) 391:811–814.
  • WARRELL RP JR, HE LZ, RICHON V, CALLEJA E, PANDOLFI, PP: Therapeutic targeting of transcription in acute pro-myelocytic leukaemia by use of an inhibitor of histone deacetylase. j Natl. Cancer Inst. (1998) 90:1621–1625.
  • ••APL is again the vanguard for a new molecular therapeuticmodality targeting histone deacetylation and gene transcrip-tion (see also [80, 811).
  • GELMETTI V, ZHANG J, FANELLI M, MINUCCI S, PELICCI PG, LAZAR MA: Aberrant recruitment of the nuclear re-ceptor corepressor-histone deacetylase complex by the acute myeloid leukaemia fusion partner ETO. Mol. Cell Biol. (1998) 18:7185–7191.
  • LUTTERBACH B, WESTENDORF JJ, LINGGI B et al.: ETO, a target of 08;21) in acute leukaemia, interacts with the N-CoR and mSin3 corepressors. Mol. Cell Biol. (1998) 18:7176–7184.

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