OncohematologyOncohematologyОнкогематология1818-83462413-4023Publishing House ABV Press67310.17650/1818-8346-2008-0-1-2-34-38HEMATOLOGIC MALIGNANCIES: DIAGNOSIS, TREATMENT, SUPPORTIVE CAREГЕМОБЛАСТОЗЫ: ДИАГНОСТИКА, ЛЕЧЕНИЕ, СОПРОВОДИТЕЛЬНАЯ ТЕРАПИЯResults of differential treatment of children with juvenile myelomonocytic leukemia with 13-cis retinoic acid and low dose cytosine-arabinosideДифференцировочная терапия с использованием 13-цис-ретиноевой кислоты и низких доз цитозин-арабинозида у детей с ювенильным миеломоноцитарным лейкозомKhachatrianL. A.ХачатрянЛ. А.
Russian Federation

Москва

MaschanM. A.МасчанМ. А.
Russian Federation

Москва

SamochatovaYe. V.СамочатоваЕ. В.
Russian Federation

Москва

SchneiderM. M.ШнейдерМ. М.
Russian Federation

Москва

BaidildinaD. D.БайдильдинаД. Д.
Russian Federation

Москва

SolopovaG. G.СолоповаГ. Г.
Russian Federation

Москва

SuntzovaYe. V.СунцоваЕ. В.
Russian Federation

Москва

ZjarikovaL. I.ЖариковаЛ. И.
Russian Federation

Москва

PetrovaU. N.ПетроваУ. Н.
Russian Federation

Москва

SinitsinaV. V.СиницынаВ. В.
Russian Federation

Москва

NovichkovaG. A.НовичковаГ. А.
Russian Federation

Москва

MaschanA. A.МасчанА. А.
Russian Federation

Москва

Federal State Enterprise «Federal Research Clinical Center of Pediatric Hematology, Oncology and Immunology of Russian Public HealthФГУ «Российская детская клиническая больница»Federal State Enterprise Russian Pediatric Clinical HospitalФГУ «Федеральный научно-клинический центр детской гематологии, онкологии и иммунологии» РосздраваFederal State Enterprise «Federal Research Clinical Center of Pediatric Hematology, Oncology and Immunology of Russian Public HealthФГУ «Российская детская клиническая больница»230520081-2343823112022231120222008https://oncohematology.abvpress.ru/ongm/article/view/673

Prognosis of patients with juvenile myelomonocytic leukemia not undergoing allogeneic hematopoietic cells transplantation (SCT) is dismal and not affected by any cytotoxic chemotherapy. Differentiating therapy is an attractive approach in JMML, due to ability of JMML cells to differentiate and die under influence of treatment with various agents in vitro. We report results of first-line treatment of 14 patients with JMML with 13-cis retinoic acid (RA) 100 mg/m2 daily peroral intake and low dose Ara-C - 25 mg/m2 daily for 10 days monthly. Complete remission (CR) or hematological improvement (HI) was obtained in 7 patients (50%). Four patients (median age at disease manifestation 5 months, median level of HbF —6,2%) achieved CR after a median of 9 cycles of therapy; in 3 patients the best response was HI (age at disease manifestation - 5; 7 and 46 months, HbF — 4,2; 8,0 and 51,7%) after 2; 3 and 5 cycles. Three out of 4 patients with CR are alive and have been in CR for 10; 17 and 30 months after start of therapy, acute lymphoblastic leukemia developed in one patient after 84 months of CR. Two patients with HI are still receiving treatment and have been in stable condition for 10 months after the initiating therapy, while 1 patient died after 8 months of therapy from catheter-related sepsis. The progression of the disease despite the treatment was registered in 7 patients (median age at disease manifestation 16,3 months, median HbF — 35,4%). We conclude that 13-cis RA in combination with low dose AraC may be useful in the treatment of patients younger than 1 year with low level of HbF, although the general paradigm of treatment of JMML with SCT remains unchanged.

Прогноз пациентов с ювенильным миеломоноцитарным лейкозом (ЮММЛ), не получивших аллогенную трансплантацию гемопоэтических клеток, остается неудовлетворительным. Одним из перспективных подходов является использование дифференцировочной терапии. В данной работе мы анализируем результаты лечения 14 пациентов с ЮММЛ, получивших лечение 13-цис-ретиноевой кислотой (100 мг/м2 ежедневный пероральный прием) и низкими дозами цитозин-арабинозида (25 мг/м2 ежемесячные 10-дневные курсы) в качестве терапии первой линии. Семь пациентов (50%) достигли полной ремиссии (ПР) или клинико-гематологического улучшения (ГУ), у 7 — заболевание прогрессировало. ПР достигнута у 4 пациентов (средний возраст на момент дебюта заболевания 5 месяцев, средний уровень HbF- 6,2 %) в среднем после проведения 9 курсов терапии, ГУ — у 3 пациентов (возраст на момент дебюта ЮММЛ: 5; 6,5 и 46 мес, HbF - 4,2; 8 и 51,7%) через 2; 3 и 5 курсов терапии. Из 4-х пациентов с ПР трое продолжают находиться в состоянии ПР в течение 30, 17 и 10 мес от начала терапии, у одного — развился ОЛЛ через 84 месяца от достижения ПР. Из трех пациентов с ГУ двое живы и продолжают лечение через 10 мес от начала терапии, 1 умер от катетер-ассоциированного сепсиса. У остальных 7 пациентов (средний возраст на момент дебюта заболевания — 16,3 месяца, средний уровень HbF- 35,4%) отмечена прогрессия заболевания. Дифференцировочная терапия 13-цис-ретиноевой кислотой и низкими дозами цитозин-арабинозида эффективна у пациентов раннего возраста с низкими уровнями HbF.

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