Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia

被引：25

作者：

Goronkova, Olga ^{[1
,13
]}

Novichkova, Galina ^{[1
]}

Salimova, Tatiana ^{[1
]}

Kalinina, Irina ^{[1
]}

Baidildina, Dina ^{[1
]}

Petrova, Ulyana ^{[1
]}

Antonova, Kristina ^{[1
]}

Sadovskaya, Maria ^{[1
]}

Suntsova, Elena ^{[1
]}

Evseev, Dmitry ^{[1
]}

Matveev, Victor ^{[1
]}

Venyov, Dmitry ^{[1
]}

Khachatryan, Lili ^{[1
]}

Litvinov, Dmitry ^{[1
]}

Pshonkin, Alexey ^{[1
]}

Ovsyannikova, Galina ^{[1
]}

Kotskaya, Natalia ^{[1
]}

Gobadze, Darina ^{[1
]}

Olshanskaya, Yulia ^{[1
]}

Popov, Alexander ^{[1
]}

Raykina, Elena ^{[1
]}

Mironenko, Olga ^{[1
]}

Voronin, Kirill ^{[1
]}

Purbueva, Bazarma ^{[2
]}

Boichenko, Elmira ^{[3
]}

Dinikina, Yulia ^{[4
]}

Guseynova, Evgeniya ^{[5
]}

Sherstnev, Dmitry ^{[6
]}

Kalinina, Elena ^{[7
]}

Mezentsev, Sergey ^{[8
]}

Streneva, Olga ^{[9
]}

Yudina, Natalia ^{[10
]}

Plaksna, Olga ^{[11
]}

Erega, Elena ^{[12
]}

Maschan, Michael ^{[1
]}

Maschan, Alexey ^{[1
]}

机构：

[1] Dmitry Rogachev Natl Med Res Ctr Pediat Hematol On, Moscow, Russia

[2] Pirogov Russian Clin Childrens Hosp, Moscow, Russia

[3] St Petersburg Childrens City Hosp 1, St Petersburg, Russia

[4] Almazov Natl Med Res Ctr, St Petersburg, Russia

[5] Krasnoyarsk Reg Clin Hosp, Krasnoyarsk, Russia

[6] Razumovsky Saratov State Med Univ, Shustov Univ Clin Hosp 3, Saratov, Russia

[7] Samara Reg Clin Childrens Hosp, Samara, Russia

[8] Perm Reg Clin Hosp, Perm, Russia

[9] Ekaterinburg Reg Clin Childrens Hosp, Ekaterinburg, Russia

[10] Voronezh Reg Clin Childrens Hosp 1, Voronezh, Russia

[11] Reg Clin Childrens Hosp, Nizhnii Novgorod, Russia

[12] Piotrovich Reg Clin Childrens Hosp, Khabarovsk, Russia

[13] Dmitry Rogachev Natl Med Res Ctr Pediat Hematol On, Dept Pediat Hematol & Oncol, Samory Mashela Str 1, Moscow 117997, Russia

来源：

BLOOD ADVANCES | 2023年 / 7卷 / 06期

关键词：

PAROXYSMAL-NOCTURNAL HEMOGLOBINURIA; RABBIT ANTITHYMOCYTE GLOBULIN; THERAPY; CYCLOSPORINE; HEMATOPOIESIS; HORSE;

D O I：

10.1182/bloodadvances.2021006716

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

We compared the efficacy and safety of eltrombopag (ELTR) combined with immunosuppressive therapy (IST) and IST alone in treatment-naive children with severe (SAA) and very severe (vSAA) aplastic anemia. Ninety-eight pediatric patients were randomized to receive horse antithymocyte globulin (hATG) and cyclosporin A (CsA) with (n = 49) or without (n = 49) ELTR. The primary endpoint was the overall response rate (ORR) at 4 months. After 4 months, nonresponders were crossed over to the alternative group. In all patients, the ORR in ELTR + IST and IST groups was similar (65% vs 53%; P = .218); however, the complete response (CR) rate was significantly higher in the ELTR + IST group (31% vs 12%; P = .027). In severity subgroups, the ORR was 89% vs 57% (P = .028) in favor of IST + ELTR in SAA, but it did not differ in patients with vSAA (52% vs 50%; P = .902). At 6 months after the crossover, 61% of initial ELTR(-) patients achieved a response compared with 17% of initial ELTR(+) patients (P = .016). No significant difference in ELTR + IST and IST groups was observed in the 3-year overall survival (OS) (89% vs 91%; P = .673) or the 3-year event-free survival (EFS) (53% vs 41%; P = .326). There was no unexpected toxicity related to ELTR. Adding ELTR to standard IST was well tolerated and increased the CR rate. The greatest benefit from ELTR combined with IST was observed in patients with SAA but not in those with vSAA. The second course of IST resulted in a high ORR in initial ELTR(-) patients who added ELTR and had limited efficacy among patients who received ELTR upfront. This trial was registered at Clinicaltrials.gov as #NCT03413306.

引用

页码：953 / 962

页数：10

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