Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation.
Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation.
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DOI：
10.1177/2040620720961910
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发表时间：
2020
影响因子：
3.4
通讯作者：
Busca A
中科院分区：
文献类型：
--
作者：
Aydin S;Dellacasa C;Manetta S;Giaccone L;Godio L;Iovino G;Bruno B;Busca A
移植物功能差或原发性移植物失败导致的移植后细胞减少患者预后差，死亡率高，主要原因是移植物抗宿主病（GVHD）、感染和/或出血。治疗选择很少，可能需要CD34+干细胞增强或第二次骨髓移植来恢复足够的造血功能。在本研究中，原发性移植物失败（n = 1）和难治性移植物功能差（n = 11）的患者在单一中心使用电子波帕治疗。三系细胞减少6例，双系细胞减少3例，单系细胞减少3例。Eltrombopag在造血干细胞移植（HCST）后214天（范围120-877）开始使用，给药时间中位数为114天（范围12天至490天）。在8/12例患者中，以75 mg/天的剂量引入，然后在1周后增加到150 mg/天；1例患者给予50 mg / d， 3例患者给予75 mg / d。在10/12例患者中，埃曲波帕显著提高了血细胞计数值，患者不再需要输血。一旦获得稳定的血液学反应，治疗逐渐减少，直到9/10有反应的患者最终停药。未见3级或4级毒性反应。最后一次随访时，3/12例患者死亡，2例因疾病复发，1例因GVHD和肺炎。除1名患者外，所有患者均保持完全缓解，并在中位858天（范围：429-1119）内保持输血独立。这些初步数据证实，eltrombopag能够挽救异基因造血干细胞移植后难治性细胞减少患者的多系造血功能。
Patients with post-transplant cytopenias due to poor graft function or primary engraftment failure show poor prognosis with a high mortality rate mainly because of graft versus host disease (GVHD), infection and/or bleeding. Treatment options are scarce and a CD34+ stem cell boost or a second bone marrow transplantation may be required to restore adequate haematopoiesis. In the present study patients with primary engraftment failure (n = 1) and refractory poor graft function (n = 11) were treated with eltrombopag in a single centre. The reason for eltrombopag treatment was trilineage cytopenia in six patients, bilineage cytopenia in three patients and single lineage cytopenia in three patients. Eltrombopag was initiated at a median of 214 (range: 120–877) days after haematopoietic stem cell transplantation (HCST) and administered for a median time of 114 (range: 12 days to >490) days. In 8/12 patients eltrombopag was introduced at a dose of 75 mg/day and then increased to 150 mg/day after 1 week; 1 patient was given 50 mg eltrombopag per day, and 3 patients received 75 mg daily. In 10/12 patients eltrombopag significantly enhanced blood count values and patients became transfusion independent. Once stable haematological response was obtained, treatment was tapered until final discontinuation in 9/10 responding patients. No grade 3 or 4 toxicities were observed. At time of last follow up, 3/12 patients were dead, 2 due to disease relapse, 1 due to GVHD and pneumonia. All patients except one maintained their complete response and remain transfusion independent at a median of 858 (range: 429–1119) days. These preliminary data confirm that eltrombopag is able to rescue multilineage haematopoiesis in patients with treatment-refractory cytopenias after allogeneic HSCT.
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艾曲波帕和改善难治性再生障碍性贫血的造血功能。
DOI：
10.1056/nejmoa1200931
发表时间：
2012-07-05
期刊：
The New England journal of medicine
影响因子：
--
作者：
Olnes MJ;Scheinberg P;Calvo KR;Desmond R;Tang Y;Dumitriu B;Parikh AR;Soto S;Biancotto A;Feng X;Lozier J;Wu CO;Young NS;Dunbar CE
通讯作者：
Dunbar CE
影响因子：
4
作者：
Chou, Fu-Sheng;Mulloy, James C.
通讯作者：
Mulloy, James C.
影响因子：
4.3
作者：
Busca, Alessandro;Dellacasa, Chiara;Bruno, Benedetto
通讯作者：
Bruno, Benedetto
影响因子：
4.8
作者：
Olsson, R.;Remberger, M.;Ringden, O.
通讯作者：
Ringden, O.
影响因子：
23.9
作者：
Qian, Hong;Buza-Vidas, Natalija;Jacobsen, Sten Eirik W.
通讯作者：
Jacobsen, Sten Eirik W.