伊马替尼联合供者淋巴细胞输注治疗造血干细胞移植后慢性粒细胞白血病复发

目的 探讨伊马替尼联合供者淋巴细胞输注(DLI)治疗异基因造血干细胞移植后慢性粒细胞白血病(CML)复发的效果.方法 3例CML(慢性期)患者,在接受预处理后,例1接受其胞妹外周血造血干细胞移植,例2接受其胞兄的骨髓移植,例3接受其胞弟的骨髓与外周血造血干细胞联合移植.例1移植后采用环孢素A(CsA)和霉酚酸酯(MMF)预防移植物抗宿主病(GVHD),例2采用CsA、短程甲氨蝶呤(MTX)、抗胸腺细胞球蛋白及抗CD25单克隆抗体预防GVHD,例3应用CsA、MTX和MMF预防GVHD.采用细胞遗传学及荧光原位杂交技术动态监测治疗效果.移植后发生血液学复发时,给予伊马替尼口服,并行DLI.结果 例1移植后30 d行DLI,输注CD3+T淋巴细胞0.5×107 /kg,移植后50 d和70 d,再次行DLI,分别输注CD3+ T淋巴细胞1.0 × 107 /kg和2.0×107 /kg,短串联重复序列(STR)检测提示为完全供者嵌合(DC).移植后120 d,疾病进展,给予伊马替尼400 mg/d,同时输注供者CD3+ T淋巴细胞2.5 × 107/kg.移植后180 d,STR检查提示仍为DC.患者最终于移植后17个月因髓外复发死亡.例2的染色体核型于移植后35 d转变为46,XY,XY为100%,BCR-ABL融合基因阴性.移植后100 d,原发病复发.停用免疫抑制剂,输入供者CD3+ T淋巴细胞3.9×107 /kg,同时口服伊马替尼500 mg/d.DLI联合伊马替尼治疗后30 d,患者的染色体核型为46,XY,XY为100%,BCR-ABL融合基因阴性,患者至今无病存活53个月.例3移植后5 d造血功能获得重建,移植后60 d,染色体核型为46,XY.移植后120 d,确诊CML复发,遂给予伊马替尼400 mg/d,并行DLI,共输注供者CD3+ T淋巴细胞8×107 /kg,1个月后,患者的染色体核型再次转为46,XY,患者至今无病存活50个月.结论 伊马替尼联合DLI对造血干细胞移植后CML复发具有一定的治疗效果.     

强化预处理allo-HSCT联合伊马替尼治疗费城染色体阳性ALL八例

目的 总结强化预处理异基因造血干细胞移植(allo-HSCT)联合伊马替尼治疗费城染色体阳性(Ph+)急性淋巴细胞白血病(ALL)的经验.方法 接受同胞allo-HSCT的Ph+ALL患者8例,移植前均达完全缓解(CR),其中5例在移植前后使用伊马替尼,3例未使用.8例中,7例采用以白消安+环磷酰胺(BuCy2)为基础的增强预处理方案,1例采用全身放疗(TBI)+Cy的增强预处理方案.患者输注单个核细胞的中位数为6.02×108/kg,输注的CD34+细胞的中位数为3.14×106/kg.术后采用环孢素A(CsA)及甲氨蝶呤(MTX)预防移植物抗宿主病(GVHD).结果 allo-HSCT后所有患者均达到白细胞植入和血小板植入,白细胞植入时间中位数为15.5 d,血小板植入时间中位数为19d;allo-HSCT后30 d,8例患者经检测均为完全供者型.患者对预处理方案的耐受性良好,未发生严重预处理相关并发症.8例患者中,4例患者发生急性GVHD,其中Ⅰ度2例,Ⅱ度1例,Ⅳ度1例.7例存活100 d以上的患者中,3例发生慢性GVHD.随访结束时共6例患者存活,其中3例无白血病存活,3例复发.死亡2例,1例死于原发病复发,1例死于急性GVHD.结论 强化预处理allo-HSCT联合伊马替尼是治疗Ph+ALL的有效方法,但在应用过程中应注意伊马替尼的抗慢性GVHD作用.

Abstract：

Objective To evaluate the outcome of combination of intensive preconditioning regimen allo-HSCT with imatinib for treatment of Ph chromosome positive acute lymphocyte leukemia (ALL). Methods Between 2009 and 2010, 8 patients diagnosed as Ph+ ALL received allo-HSCT from HLA identical sibling during complete remission. Imatinib was added into the therapies of 5 patients.Seven patients received the intensive preconditioning regimen based on BuCy2, one patient received the regimen of TBI-Cy. A median of 6. 02 × 108/kg mononuclear cells and 3. 14 × 106/kg CD34+ cells were transfused. GVHD prophylaxis included cyclosporine A and methotrexate. Results All patients were well tolerant to the regimen without serious regimen-related toxicity. The median time of ANC≥0. 5 × 109/L was 15. 5 days, and that of PLT≥20 × 109/L was 19 days. Thirty days after allo-HSCT, all patients got donor engraftment successfully. Among 8 cases, 4 cases presented acute GVHD, 2 developed degree Ⅰ , one developed degree Ⅱ , and one developed degree Ⅳ. Seven patients were alive 100 days after allo-HSCT, 3 of whom presented chronic GVHD. At the end of following-up period, 6 patients were alive, among them, 3 patients were alive without relapse; 3 patients relapsed; Two patients died, one from acute GVHD, and one from leukemia relapse. Conclusion Combined intensive preconditioning regimen allo-HSCT with Imatinib was an effective treatment for Ph+ ALL, but the effect of anti-chronic GVHD of imatinib should arouse certain attention.     

3例造血干细胞移植术后并发重度肠道移植物抗宿主病患者的护理

对2例慢性髓性白血病和1例急性髓性白血病患者行异因因造血干细胞移植(allo-HSCT).干细胞分别来自血缘HLA相合、非血缘HLA相合及血缘HLA 1个位点不合的供者.3例患者于移植后60、105、116 d并发重度肠道移植物抗宿主病(GVHD),予以免疫抑制剂治疗及相应的护理措施,结果1例因肺部结核杆菌及白色念珠菌感染死亡,2例得到有效控制.提示allo-HSCT后并发重度肠道GVHD进展快、症状重,密切观察病情,加强消化道护理、心理护理、保护性隔离及用药护理对移植患者的预后至关重要.     

3例造血干细胞移植术后并发重度肠道移植物抗宿主病患者的护理

对2例慢性髓性白血病和1例急性髓性白血病患者行异因因造血干细胞移植（allo-HSCT）。干细胞分别来自血缘HLA相合、非血缘HLA相合及血缘HLA1个位点不合的供者。3例患者于移植后60、105、116d并发重度肠道移植物抗宿主病（GVHD），予以免疫抑制剂治疗及相应的护理措施，结果1例因肺部结核杆菌及白色念珠菌感染死亡．2例得到有效控制。提示allo-HSCT后并发重度肠道GVHD进展快、症状重，密切观察病情。加强消化道护理、心理护理、保护性隔离及用药护理对移植患者的预后至关重要。     

异基因造血干细胞移植后早期合并肾病综合征一例

正异基因造血干细胞移植(allogeneic hematopoietic stem cell transplantation,allo-HSCT)广泛用于治疗血液系统疾病,随着移植技术的不断进步,allo-HSCT成功率逐步提高。然而,移植后并发症如急、慢性移植物抗宿主病(graft-versus-host disease,GVHD)、肾脏损害、阻塞性细支气管炎伴机化性肺炎等,仍严重影响患者术后生活质量甚至威胁生命。Allo-HSCT后肾脏损害较少见,以肾病综合征(nephrotic     

异基因造血干细胞移植后带状疱疹的临床分析

目的 探讨异体基因造血干细胞移植(allo-HSCT)后带状疱疹的发病情况、临床特点、诊治及预后.方法 对河南省肿瘤医院血液科allo-HSCT受者带状疱疹的发生时间、临床表现、治疗及预后进行回顾性分析.结果 可评估的191例接受allo-HSCT患者中有18例并发带状疱疹,发病率为9.42%,其中14例发生在干细胞移植治疗后3～12个月内.10例合并慢性移植物抗宿主病(GVHD),1例合并急性GVHD.临床表现主要为区域性皮肤疱疹.3例出现严重疱疹后神经痛,需强效止痛.无泛发性疱疹及内脏受累病例.所有患者接受抗病毒药物阿昔洛韦和阿糖腺苷静脉输注治疗,全部治愈,无疱疹相关死亡患者,平均治疗时间为15.5 d.结论 带状疱疹是allo-HSCT后常见的病毒感染性疾病,主要发生在干细胞移植治疗后3～12个月,GVHD是其发生的高危因素.阿昔洛韦和阿糖腺苷的疗效较好.     

慢性眼移植物抗宿主病的临床特点及相关机制的新进展

异基因造血干细胞移植(allo-HSCT)可有效治疗多种血液病,目前随着移植技术的不断提高和经济水平的飞速发展,越来越多的血液病患者因接受了allo-HSCT治疗而得以长期存活.慢性眼移植物抗宿主病(coGVHD)是allo-HSCT术后最常见的眼部并发症,主要表现为难治性干眼,严重时可导致眼表稳态失衡、角膜缘干细胞缺...     

异基因造血干细胞移植后共刺激分子LIGHT/HVEM的表达与GVHD的关系

目的 探讨异基因造血干细胞移植(allo-HSCT)后共刺激分子LIGHT和HVEM的表达与移植物抗宿主病(GVHD)发生的关系.方法 26例allo-HSCT患者中,预处理方案采用环磷酰胺+足叶乙甙+全身照射11例,采用氟达拉滨+阿糖胞苷+白消安12例,采用环磷酰胺3例.移植时,输注单个核细胞(5.89±3.36)×10~8/kg,CD34~+细胞(3.29±1.29)×10~6/kg.预防急性GVHD(aGVHD)的方案采用环孢素A+短程甲氨蝶呤+吗替麦考酚酯,HLA半相合及非血缘关系者加用抗胸腺细胞球蛋白.GVHD的诊断与分级采用西雅图标准.在预处理前、移植后15 d、发生aGVHD时及aGVHD经治疗好转后分别采集患者外周血2 ml,采用三色流式细胞术进行检测,同时以15名健康志愿者的血液标本作为正常对照.结果 所有患者均获得造血功能重建,移植物均完全植活.移植后共发生aGVHD9例,发生率为35%,其中Ⅲ～Ⅳ度者3例,8例经治疗后好转,1例治疗无效死亡;发生慢性GVHD(cGVHD)7例,发生率为26.9%.患者预处理前和移植后15 d及正常人外周血T淋巴细胞几乎不表达LIGHT,组成性表达HVEM;发生GVHD时,LIGHT表达显著上调,HVEM表达下调;GVHD好转后,LIGHT和HVEM的表达恢复正常.移植后15 d时,发生aGVHD者LIGHT的表达高于未发生aGVHD者,而HVEM的表达较低(P<0.05).Ⅲ～Ⅳ度aGVHD者LIGHT的表达明显高于Ⅰ～Ⅱ度者(P<0.05).结论 LIGHT/HVEM在allo-HSCT后GVHD的发生和发展过程中起重要作用,通过监测该共刺激分子的表达对aGVHD的发生有一定预示作用.     

自然杀伤T淋巴细胞与移植物抗宿主病的研究进展

移植物抗宿主病(GVHD)是目前导致异基因造血干细胞移植(allo-HSCT)失败和受者死亡的重要因素,与移植物抗白血病效应(GVL)和受者的生活质量密切相关.GVHD是由供者移植物巾的T淋巴细胞识别宿主抗原引起的免疫反应所导致受者多器官损伤,涉及各种免疫活性细胞、细胞因子和炎症因子[1].     

自然杀伤T淋巴细胞与移植物抗宿主病的研究进展

移植物抗宿主病(GVHD)是目前导致异基因造血干细胞移植(allo-HSCT)失败和受者死亡的重要因素,与移植物抗白血病效应(GVL)和受者的生活质量密切相关.GVHD是由供者移植物巾的T淋巴细胞识别宿主抗原引起的免疫反应所导致受者多器官损伤,涉及各种免疫活性细胞、细胞因子和炎症因子[1].     

Pneumonia in allogeneic stem cell transplantation recipients: a multicenter prospective study

Pneumonia is a common cause of mortality after allogeneic hematopoietic stem cell transplantation (allo-HSCT) but updated and prospective information is partial. The aim of this nationwide prospective study is to determine the current epidemiology, etiology, and outcome of pneumonia in allo-HSCT recipients. From September-2003 to November-2005, 112 episodes in 427 consecutive allo-HSCT recipients were included (incidence 52.2 per 100 allo-HSCT/yr), and 72 of them (64.3%) were microbiologically defined pneumonia. Bacterial pneumonia (44.4%) was more frequent than fungal (29.2%) and viral pneumonia (19.4%). The most frequent microorganisms in each group were: Escherichia coli (n = 7, 8.9%), Streptococcus pneumoniae (n = 4, 5.0%), cytomegalovirus (n = 12, 15.4%), and Aspergillus spp. (n = 12, 15.4%). The development of pneumonia and chronic graft-versus-host disease (GVHD) was associated with increased mortality after allo-HSCT, and the probability of survival was significantly lower in patients that had at least one pneumonia episode (p < 0.01). Pneumonia development in the first 100 d after transplantation, fungal etiology, GVHD, acute respiratory failure, and septic shock were associated with increased mortality after pneumonia. Our results show that pneumonia remains a frequent infectious complication after allo-HSCT, contributing to significant mortality, and provide a large current experience with the incidence, etiology and outcome of pneumonia in these patients.     

Highlighting the interaction between immunomodulatory properties of mesenchymal stem cells and signaling pathways contribute to Graft Versus Host Disease management

BackgroundAllogeneic hematopoietic stem cell transplantation (Allo-HSCT) has been increasingly used as a therapeutic approach for hematological malignancies. Several potential strategies have been developed for treating or preventing allo-HSCT complications, specifically graft-versus-host disease (GVHD). GVHD could significantly affect the morbidity and mortality of patients after allo-HSCT. Curative treatment and prophylaxis regimens for GVHD could reduce GVHD incidence and improve survival rate. Among these therapeutic strategies, mesenchymal stem cell (MSCs) mediated immunomodulation has been explored widely in clinical trials. MSCs immunomodulation ability in GVHD correlates with the interactions of MSCs with innate and adaptive immune cells. However, signaling pathways responsible for MSCs' impact on GVHD regulation, like JAK/STAT, NOTCH, MAPK/ERK, and NFκβ signaling pathways, have not been clearly described yet. This review aims to illuminate the effect of MSCs-mediated immunomodulation in GVHD management after allo-HSCT representing the role of MSCs therapy on signaling pathways in GVHD.ConclusionMSCs could potentially modulate immune responses, prevent GVHD, and improve survival after allo-HSCT. Previous studies have investigated different signaling pathways' contributions to MSCs immunoregulatory ability. Accordingly, targeting signaling pathways components involved in MSCs related GVHD regulation is proven to be beneficial.     

RIFLE标准对慢性粒细胞白血病清髓性异基因造血干细胞移植后急性肾损伤的评价及预后分析

目的 探讨慢性粒细胞白血病清髓性异基因造血干细胞移植（HSCT）后急性肾损伤（AKI）的发生率和危险因素及其对患者移植后6个月生存率的影响。 方法 应用RIFLE标准对93例慢性粒细胞白血病患者清髓性异基因HSCT后肾脏功能的变化情况进行回顾性分析。 结果 清髓性异基因HSCT后100 d内有39例（41.9%）患者发生AKI,其中AKI危险（AKI-R）24例（25.8%）,AKI损伤（AKI-I）10例（10.8%）,AKI功能衰竭（AKI-F）5例（5.4%）,中位时间为干细胞回输后40 d（1～96 d）。移植后发生≥Ⅲ度急性移植物抗宿主病（aGVHD）患者与<Ⅲ度aGVHD患者100 d内AKI发生率分别为（81.82±11.63）%和（36.59±5.32）%（P ＝ 0.0037）。移植后出现总胆红素增高患者与无增高患者100 d内AKI发生率分别为（72.73±13.43）%和（37.04±5.37）%（P ＝ 0.0192）。移植后发生≥Ⅲ度aGVHD是患者发生AKI的独立危险因素,其相对危险度（RR）为2.773[95%可信区间（CI）（1.073～7.167）,P ＝ 0.035];并且移植后发生≥Ⅲ度aGVHD患者发生AKI-I和AKI-F的RR为6.320[95%CI（1.464～27.291）,P ＝ 0.013]。移植后发生AKI患者100 d内病死率与无AKI患者差异有统计学意义（P ＝ 0.001）。移植后发生AKI-R、AKI-I和AKI-F的患者6个月的生存率分别为（86.96±7.02）%、（70.0±14.49）%和0（P ＝ 0.000）。 结论 AKI是慢性粒细胞白血病清髓性异基因HSCT后的重要并发症。移植后出现≥Ⅲ度aGVHD和总胆红素增高是发生AKI的影响因素。出现≥Ⅲ度aGVHD的患者易发生较重的AKI。移植后发生AKI程度越严重,患者6个月的生存率越低。RIFLE标准能提高早期诊断AKI的敏感性,并可监测肾功能进展情况,预测预后。     

Post-transplant cyclophosphamide and early mixed donor Chimerism in myeloid malignancies; a single-center experience

BackgroundAllogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative option for high-risk myeloid malignancies. Post-transplant cyclophosphamide (PT-Cy) has proven to be effective for graft versus host disease (GVHD) prophylaxis. Given that graft-versus-tumor (GVT) effect plays a major role in reducing the risk of disease relapse, the application of PT-Cy must balance the risk of relapse. Mixed chimerism (MC) refers to a state of concurrent presence of recipient and donor cells post allo-HSCT which may precede relapse disease.ObjectiveWe investigated the impact of PT-Cy on early MC (EMC) and disease relapse in patients with a myeloid malignancy post allo-HSCT.Study designThis retrospective single-center study included patients that underwent allo-HSCT between 2015 and 2021. Patient and disease characteristics were collected from the electronic health records. EMC was defined as <95% donor cells at day 90–120 post allo-HSCT.ResultsA total of 144 patient that received an allo-HSCT were included in the study. One hundred and eight (75%) patients received PT-Cy as part of the GVHD prophylaxis regimen. The majority underwent allo-HSCT for acute myeloid leukemia (62%) or myelodysplastic syndrome (31%). Sixty-five percent received allo-HSCT from a matched unrelated donor transplant and 65% received a myeloablative conditioning regimen. A lower rate of chronic GVHD (p = 0.03) and a higher rate of EMC (p = 0.04) were observed in patients that received PT-Cy. PT-Cy was not associated with overall survival (OS) and relapse-free survival (RFS). Multivariable analysis identified measurable residual disease status (p = 0.003), hematopoietic cell transplantation-specific comorbidity index (p = 0.012) and chronic GVHD (p = 0.006) as independent prognostic variables for OS. AML-adverse risk (p = 0.004) and EMC (p = 0.018) were independently prognostic for RFS. While EMC overall was not significantly associated with higher risk of relapse, EMC was associated with shorter RFS within adverse-risk AML patients.ConclusionOur study shows that PT-Cy was associated with an increased risk of EMC. The predictive value of EMC for relapse remains unclear and may depend on the underlying disease, which should be validated in a larger cohort.     

High expression of granzyme B in conventional CD4+ T cells is associated with increased relapses after allogeneic stem cells transplantation in patients with hematological malignancies

Granzyme B is known to be a serine protease contained in granules of cytotoxic T cells. We have previously reported an influence of granzyme B expression in T regulatory cells (Tregs) on the risk of acute graft versus host disease (GVHD) onset. However, it is still unknown if conventional T cells (Tcon) use the granzyme B pathway as a mechanism of alloimmunity. We hypothesized that granzyme B in Tcon may affect recurrence within the first 6 months after allogeneic transplantation (allo-HSCT). A total of 65 patients with different hematological malignancies were included in this study. Blood samples were collected on day +30 after allo-HSCT. The percentage of granzyme B positive conventional T cells in patients who developed relapse in the first 6 months after allo-HSCT was 11.3 (4.5–35.3) compared to the others in continuous complete remission—1.3 (3.65–9.7), р = 0.011. The risk of relapse after allo-HSCT was in 3.9 times higher in patients with an increased percentage of granzyme B positive conventional T cells. The findings demonstrated that the percentage of granzyme B positive conventional T cells on day +30 after allo-HSCT could be a predictable marker of relapse within the first 6 months after allo-HSCT.     

The response-guided ATG treatment provides a survival benefit and KPS recovery for patients with steroid refractory acute GVHD: The Nagasaki Transplant Group Experience

Steroid-refractory acute graft-versus-host disease (SR-aGVHD) is a serious complication that negatively affects the prognosis and quality of life of patients who receive allogeneic hematopoietic stem cell transplantation (allo-HSCT). Antithymocyte globulin (ATG) is one of the second-line treatments for SR-aGVHD. We retrospectively evaluated Karnofsky Performance Status (KPS) recovery and clinical response in 11 patients who received the response-guided low-dose ATG treatment for SR-aGVHD after allo-HSCT using alternative donors. The median dose of ATG per cycle was 1.0 mg/kg (range, 1.0–1.25 mg/kg) and the median number of cycles of ATG was 2 (range, 1–4). The overall response rate was 63.6%, and the estimated overall survival rate at 1 year was 63.6%. Two out of seven patients who survived 1 year after the response-guided ATG treatment had KPS of 80 or higher. The remaining 5 patients had KPS of lower than 80 due to moderate chronic GVHD (cGVHD) and/or ≥grade 3 infectious complications. Based on the poor prognosis of patients with SR-aGVHD, the response-guided ATG treatment represents one therapeutic option. The present results also suggest that chronic GVHD and infectious complications after the response-guided ATG treatment were associated with decreased KPS recovery and impaired social function.     

异基因造血干细胞移植治疗重型β珠蛋白生成障碍性贫血

目的 探讨异基因造血干细胞移植(allo-HSCT)治疗重型β珠蛋白生成障碍性贫血的临床疗效.方法 PesaroⅡ-Ⅲ度重型β珠蛋白生成障碍性贫血患者24例接受allo-HSCT治疗,其中男性18例,女性6例,患者年龄中位数为4岁(2～15岁).24例中,同胞供者23例,母亲供者1例;HLA 6个抗原全相合23例,5个抗原相合1例;骨髓混合外周血干细胞移植15例,脐带血移植9例.采用白消安+环磷酰胺+氟达拉滨的预处理方案.环孢素A(CsA)+甲氨蝶呤(MTX)+抗胸腺细胞球蛋白(ATG)+吗替麦考酚酯(MMF)联用预防移植物抗宿主病(GVHD).中位随访时间13个月(3～69个月).结果 移植后22例患者造血功能顺利恢复.至随访结束,无病存活21例;移植相关死亡1例(4.2%);移植物排斥反应2例(8.3%).21例的3年无病存活率为87.5%,3年总体存活率为91.7%.Ⅱ-Ⅳ度急性GVHD的累积发生率为16.7%,慢性GVHD的累积发生率为20.3%,广泛性慢性GVHD的发生率为5.0%.结论 异基因骨髓混合外周血干细胞移植治疗珠蛋白生成障碍性贫血可获得确切疗效,同时脐带血是珠蛋白生成障碍性贫血移植的重要干细胞来源.CsA+MTX+ATG+小剂量、短疗程MMF的方案可以有效地减少严重急性GVHD的发生,提高移植疗效.

Abstract：

Objective To investigate the effect of allgeneic hematopoietic stem cell transplantation (allo-HSCT) for β-thalassemia major. Methods Twenty-four β-thalassemia major patients with median age of 4 years (range: 2～15 years), 18 boys and 6 girls, received allo-HSCT.They were classified into class Ⅱ-Ⅲ according to Pesaro thalassemia classification. Twenty-three transplantations were from sibling donor and 1 was from mother, either HLA-identical (n = 23) or HLA-mismatched (5/6) (n = 1). Fifteen patients received bone marrow transplantation (BMT) plus peripheral blood stem cell transplantation (PBSCT), and 9 were subjected to umbilical cord blood transplantation (UCBT). The conditioning regimen consisted of busalphan, cyclophosphamide,fludarabine, plus hydroxyurea before transplantation. Graft-versus-host disease (GVHD) prophylaxis included CsA, methotrexate, antilymphpcute globulin, and mycophenolate mofetil. The median follow-up period was 13 months (range: 3～69). Results Of 24 patients, there were 21 cases (87. 5 %) of disease-free survival, 1 (4. 2 %) transplantation-related death, and 2 cases (8. 3 %) of rejection. Three-year overall survival and disease-free survival rate was 91.7 % and 87. 5 %respectively. The cumulative incidence of grade Ⅱ -Ⅳ acute GVHD and chronic GVHD was 16. 7 %and 20. 3 %, particularly cumulative extensive chronic GVHD was 5. 0 %. Conclusion The sibling donor BMT plus PBSCT is an effective and safe way to treat β-thalassemia major. Cord blood is an important source of hematopoietic stem cells for HSCT. The protocol GVHD prophylaxis of CsA,MTX, ATG with a low-dose and short course of MMF can effectively reduce the incidence of severe acute GVHD, improve the outcome of thalassemia transplantation.     

Long-term remission of HSCT-related NS after a second allogenic stem cell transplant

Background

Hematopoietic stem cell transplantation (HSCT)-related nephrotic syndrome (NS) is a rare event and has been described as a clinical form of chronic graft-versus-host disease (GVHD). Although immunological mechanisms are thought to play important roles in NS after HSCT, the exact mechanisms have not been clarified.

Case-Diagnosis/Treatment

We report a 4-year-old boy with acute lymphoblastic leukemia (ALL) who developed NS during the tapering of immunosuppressants 5 months after an allogeneic HSCT (allo-HSCT). A renal biopsy was performed, and light and electron microscopy revealed minimal change disease (MCD). Although the response to treatment with steroids and tacrolimus was favorable, the child experienced two relapses of NS within the first 9 months after the initial response. A second allo-HSCT was performed to treat the relapse of ALL. After the second allo-HSCT, the remission of NS was maintained without recurrence for 5 years, even after the cessation of immunosuppressants.

Conclusions

Our patient who had ALL and developed NS after his first allo-HSCT, maintained remission from NS after a second allo-HSCT. This suggests that the immune cells from the first donor origin were associated with the pathogenesis of NS.