Jasmina Hauptman, Darja Žontar, Irena Preložnik Zupan. Podčetrtek,
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1 Jasmina Hauptman, Darja Žontar, Irena Preložnik Zupan Podčetrtek,
2 Gre za življenje ogrožujočo bolezen, ki lahko vodi do kronične okvare organov ter prezgodnjo smrt, predvsem zaradi tromboz ali odpovedi ledvic! Zaradi raznolikosti simptomov in znakov, s katerimi se bolezen kaže, zdravniki še vedno preredko ter prepozno pomislimo nanjo!
3 Pridobljena somatska mutacija PIG - A gena na X kromosomu prepreči vsem GPI vezanim proteinom vezavo na celično membrano CD59 Tvori obrambno ovojnico ERI pred lizo komplementa1 CD55 Preprečuje nastanek in povečuje nestabilnost C3 konvertaze ter oslabi komplementno kaskado2 CD59 GPI-vezalci CD55 1. Johnson RJ et al. J Clin Pathol: Mol Pathol 2002;55: Brodsky R. Paroxysmal Nocturnal Hemoglobinuria. In: Hematology - Basic Principles and Practices. 4th ed. R Hoffman; EJ Benz; S Shattil et al. eds. Philadelphia, PA: Elsevier Churchill Livingstone; 2005; pp
4 Normalne rdeče celice so zaščitene pred aktivacijo komplementa ter lizo z inhibitorji Brez zaščite - GPI vezanih proteinov pride do lize celic Tromboza Odpoved ledvic Velik vpliv na preživetje Pljučna hipertenzija Aktivacija komplementa Bolečina v trebuhu Dispneja Normalne RBC Prost Hemoglobin Disfagija Utrujenost Hemoglobinurija Vpliv na kvaliteto življenja Anemija Erektilna Disfunkcija 1. International PNH Interest Group. Blood. 2005;106: Brodsky R Paroxysmal Nocturnal Hemoglobinuria. In: Hematology - Basic Principles and Practices. 4th ed. R Hoffman; EJ Benz; S Shattil et al, eds. Philadelphia, PA: Elsevier Churchill Livingstone; 2005; p Rother RP et al. JAMA. 2005;293: Socie G et al. Lancet. 1996;348: Hill A et al. Br J Haematol. 2007;137: Lee JW et al. Hematologica (s2): Abstract #505 and Hill A et al. Br J Haematol. 2010; May;149(3): Hillmen P et al. Am. J. Hematol. 2010; 85:
5 Chronic Hemolysis is the Underlying Cause of Progressive Morbidities and Mortality of PNH Thrombosis Venous Arterial PE/DVT Stroke/TIA Cerebral MI Dermal Hepatic/Portal Abdominal ischemia Fatigue / Impaired Quality of Life Abdominal pain Dysphagia Poor physical functioning Erectile dysfunction Chronic Kidney Disease Renal insufficiency Dialysis Hypertension End Organ Damage Brain Liver GI Pulmonary Hypertension Dyspnea Cardiac Dysfunction Anemia Transfusions Hemosiderosis 1. International PNH Interest Group. Blood. 2005;106: Brodsky R. Paroxysmal Nocturnal Hemoglobinuria. In: Hematology - Basic Principles and Practices. 4th ed. R Hoffman; EJ Benz;S Shattil et al, eds. Philadelphia, PA: Elsevier Churchill Livingstone; 2005; p Hillmen P et al. N Engl J Med. 1995;333: Rosse W et al. Hematology (Am Soc Hematol Educ Program). 2004: Rother R et al. JAMA. 2005;293: Socie G et al. Lancet. 1996;348: Hill A et al. Br J Haematol. 2007;137: Lee JW et al. Hematologica (s2): Abstract #505 and Hill A et al. Br J Haematol. 2010; May;149(3): Hillmen P et al. Am. J. Hematol. 2010; 85:
6 prospektivna analiza 68-ih vzorcev periferne krvi, sprejetih v specializiranem hematološkem laboratoriju UKC Ljubljana v enoletnem obdobju od do , poslanih zaradi suma na PNH večparametrična visoko specifična pretočna citometrija odvzem vzorca perifene krvi (epruveta s heparinom ali EDTA), dostava v laboratorij najkasneje v 24 urah klon PNH se določuje izključno na zrelih celicah! (pomik v levo ali nevtropenija preiskava se ne opravi)
7 Granulociti - najzaneslivejši za dokaz PNH klona, Monociti - potrjujejo PNH klon na granulocitih (klon pogosto večji, kot na granulocitih; natančnost manjša zaradi manjšega števila); Limfociti - niso primerni za določevanje klona zaradi drugačne življenjske dobe; Eritrociti - primerni za oceno klona, vendar velikost letega variira zaradi hemolize ali transfuzije;
8 Count Type III 0.0% Type II 0.0% Type I 100% Normal RBCs with normal CD59 expression (Type I cells) CD59-PE Type III 6% Type II 0.1% Type I 93.9% PNH clone with complete CD59 deficiency (Type III cells) CD59-PE CD59-PE Gating on GPA+ RBCs Type III 11.9% Type II 71.3% Type I 16.8% PNH clone with complete CD59 deficiency (Type III cells) and partial CD59 deficiency (Type II cells) CD59-PE GPA: glycophorin A 1. Borowitz MJ et al. for the International Clinical Cytometry Society. Cytometry B Clin Cytom. 2010;78B:
9 CD24-PE Granulocytes Count White blood cells (WBC) Red blood cells (RBC) Type III 6% Type II 0.1% Type I 93.9% FLAER-ALEXA GPI anchor binding marker CD59-PE GPI anchored protein 55% of Granulocytes lack GPI proteins 6% RBCs are Type III PNH cells 1. Borowitz MJ et al. for the International Clinical Cytometry Society. Cytometry B Clin Cytom. 2010;78B:
10
11 Bolnišnica Število poslanih vzorcev Število dokazanih PNH UKC Ljubljana 40 8 UKC Maribor 4 2 SB Celje 12 1 SB Novo Mesto 6 2 SB Murska Sobota 2 0 SB Slovenj Gradec 2 0 SB Izola 1 0
12 Specialnosti napotnih Število poslanih Število pozitivnih zdravnikov vzorcev vzorcev Hematolog Nefrolog 3 0 Kardiolog ter angiolog 2 0 Gastroenterolog 1 0
13 13/68 (19%) bolnikov je imelo prisoten PNH klon - prevalenca 6,5/ prebivalcev, 4/13 bolnikov so bili na novo odkriti incidenca 2/ prebivalcev, Ž/M = 9/4, povprečna starost 46,6 let, vzrok napotitve k hematologu 12/13 citopenija, 1/13 hematurija, lab. preiskave, citološka in histološka preiskava KM 5/12 bolnikov PNH klon v sklopu aplastične anemije, 6/12 MDS, 1/12 ITP
14 Simptomi in znaki ob diagnozi: - 13/13 utrujenost ter dispneja, - 2/13 temen urin zaradi hemoglobinurije, - 2/13 intermitentna ledvična insuficienca, Tekom spremljanja 1/13 bolnikov utrpel vensko trombozo, 1/13 (bolnik z MDS)prehod v akutno levkemijo, 1/13 umrl zaradi infekta ob levkopeniji (bolnik z MDS).
15 Zdravljenje: - 11/13 bolnikov imunosupresivno zdravljenje (KS, anti limfocitni imunoglobulin, ciklosporin, alemtuzumab) - 3/13 (PNH klon>50%) antikoagulantno zdravljenje, - 4/13 monoklonsko protitelo proti C5 komplementa EKULIZUMAB, - 1/13 bolnikov PKMC neuspešno, - 6/13 bolnikov je transfuzijsko odvisnih (1 enota krvi/mesečno).
16 Laboratorij Bolnik 1 Bolnik 2 Bolnik 3 Bolnik 4 Spol Ž M M Ž Starost (leta) Simptomi in znaki Utrujenost Utrujenost, Utrujenost, Utrujenost dispneja dispneja - Konc. Hb (g/l) - Število Tr (x10 9 ) - Število L (x10 9 ) - Retikulociti (x10 9 ) - Bilir cel (µmol/l) - Bilir dir (µmol/l) - LDH (µkat/l) , , ,6 0, , ,3 1, , ,9 44, ,51 Klon PNH (%): -Granulocit -Monociti. -Eritrociti tip II+III Pridružena bolezen kostnega mozga 19,2 17,0 3,0 2,6 3,8 0,2 1,2 2,70 0,40 21,2 22,5 3,5 MDS MDS AA MDS Odvisnost od transfuzij Ne Da Da Ne Zdravljenje IST / IST IST
17 PNH bolniki Število znanih bolnikov, spol (M/Ž) 9 (6/3) Povprečna starost (leta) 41,5 Laboratorij (povprečne vrednosti) - Koncentracija Hb (g/l) - Število Tr (x10 9 ) - Število L (x10 9 ) - Število retikulocitov (x10 9 ) - Bilirubin celokupni (µmol/l) - Bilirubin direktni (µmol/l) - LDH (µkat/l) Povprečna vrednost klona PNH (%) - Granulociti - Monociti - Eritrociti (tip II+III) 110,3 95,2 3,65 108,7 29,3 9,8 6,55 51, ,4
18 Klasična PNH Pridružena bolezen kostnega mozga (MDS/AA) ITP PNH bolniki 1 3 / 4 1 Simptomi in znaki ob diagnozi: Utrujenost 9 Hemoglobinurija 2 Dispneja 2 Ledvična odpoved 2 Bolečine v trebuhu 0 Tromboza 0 Odvisnost od transfuzij (Da/Ne) 4/5 Zdravljenje: Imunosupresivno Ekulizumab PKMC Antikoagulantno
19 Število 55 Spol (M/Ž) 34/21 Starost 52,4 Vzrok napotitve: Citopenija Znaki hemolize Nepojasnjena tromboza Hemoglobinurija Avtoimuno dogajanje Neznano Klon PNH odsoten
20 Število bolnikov Velikost PNH klona (%)
21 PODSKUPINE PNH KLASIČNA 1/13 SUBKLINIČNA 7/13 V SKLOPU DRUGE BOLEZNI KOSTNEGA MOZGA 5/13 INTRAVASKULARNA HEMOLIZA huda Brez kliničnega ali biokemičnega dokaza za hemolizo BOLEZEN KOSTNEGA MOZGA (AA/MDS) Normalna morfologija KM prisotna PRETOČNA CITOMETRIJA Velik PNH klon (>50%) Majhen PNH klon Blaga do srednja prisotna Variabilno, navadno manjši klon (<30%) TERAPIJA Z EKULIZUMABOM Da Ne Odvisno od velikosti klona
22 KDAJ POMISLITI NA PNH? Pridobljena hemolitična anemija in znaki hemolize (Coombsovi testi negativni) Laboratorijski znaki intravaskularne hemolize (hemoglobinemija, hemoglobinurija, hemosiderinurija, zvečana raven LDH, zmanjšana vrednost plazemskega haptoglobina) Citopenije (granulocitopenija in/ali trombocitopenijain/ali anemija) ob zvečanem številu retikulocitov ali znakih hemolize Nepojasnjene venske tromboze (predvsem abdominalnih ali cerebralnih ven) Aplastična anemija, mielodisplastični sindrom, predvsem ob kliničnih ali laboratorijskih znakih hemolize Epizodična disfagija ali abdominalna bolečina z znaki hemolize Določi PNH klon Specialistični hematološki laboratorij KOH, UKC Ljubljana Odvzem venske krvi, epruveto z EDTA (5 ml), pošiljaj pri sobni temperaturi, do 24 ur od odvzema.
23 HVALA ZA POZORNOST!!!
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