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Patient | Diagnosis | Frontline therapy | Therapy of relapse | Time from last GO to hemolysis | Infections | Symptoms during hemolysis | Outcome | Publication |
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Patient 1 : 21-year-old female (index patient) | Relapsed CBF-AML (BM-blasts 65%) t (8; 12), corresponding RUNX1/RUNX1T1-fusion gene. Additional mutations in PHF6, EZH2, and KDM6A. No extramedullary disease. | 7 + 3 (cytarabine + daunorubicin), 3 x HD-AraC. Relapse after 2 mo. | 7 + 3 (cytarabine and idarubicin) and GO (3 mg/m2, days 1, 4, 7) | 19 days | Enterococcus faecium sepsis, Clostridium difficile colitis, HHV6 viremia, HSV stomatitis | Fever, convulsions, confusion, mild diastolic hypertension | Recovered after repeated TPEs. Response to therapy: CRi, MRD-neg | Current |
Patient 2 : 24-year-old female | Primary CBF-AML (BM-blasts 69%) Inv16, corresponding CBFB-MYH11-fusion gene. Axillary and inguinal lymphadenopathy. | 7 + 3 (cytarabine and idarubicin) and GO (3 mg/m2 on day 4) | — | 14 days | Suspected infection, blood cultures negative and virus samples negative. No deep foci of infection detected. | Fever, hypertension, bradycardia | Recovered after 3 TPEs. Response to therapy: CR, MRD 0.02% | Current |
2-year-old boy | Relapsed AML M4/M5 | NOPHO-AML93 + MUD-SCT. Relapse 18 mo after SCT. | FLAG and 2 doses of GO (7.5 mg/m2) at a 2-week interval | 3 weeks | Staphylococcal sepsis | Abdominal pain, hypertension, persistent high fever, malaise | Recovery, but died later from PD | Maniecki et al. Blood 2000 [7] |
1-year-old girl | Relapsed AML M5 | NOPHO-AML 2004 with 2 doses of GO (5 mg/m2, 2-week interval). Relapse after 3 mo. | FLAG-DaunoXome, 2 doses of GO (6 mg/m2, 2-week interval) and NOPHO-AML 2004 induction | 5 weeks | Suspected infection, blood cultures negative | Hypertension, confusion, convulsions | Did not recover from hemolytic episode and died from PD 72 days from the first GO | Maniecki et al. Blood 2000 [7] |
12-year-old boy | Refractory AML | Relapse after two salvage chemotherapies | GO on days 1 and 15, dose not reported | Hemolytic samples from day 5 of GO cycle | Vancomycin-resistant Enterococcus faecium and Trichosporon asahii infections | Hypertension, multiorgan failure and need for renal replacement therapy | Did not recover from hemolysis and died 40 days after the first GO | Tesfazghi et al. Clin Chem 2018 [8] |
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