Unidad Pediátrica De Hematología, Oncología y Trasplante Hematopoyético, Hospital De La Santa Creu i Sant Pau, Barcelona, Spain.
Unidad Pediátrica De Hematología, Oncología y Trasplante Hematopoyético, Hospital De La Santa Creu i Sant Pau, Barcelona, Spain.
An Pediatr (Engl Ed). 2022 Aug;97(2):103-111. doi: 10.1016/j.anpede.2021.08.010. Epub 2022 Jul 20.
Hypogammaglobulinemia in the first months after allogeneic hematopoietic stem cell transplantation (HSCT) is common in paediatric patients. During this phase, replacement therapy with human immunoglobulin must be administered parenterally to prevent infections. In some cases, this hypogammaglobulinemia persists over time, which forces further treatment when the patient is usually no longer a carrier of a central line, making them ideal candidates for subcutaneous replacement therapy. There is little published literature describing the use of this method in paediatric patients undergoing HSCT, widely described in replacement treatment in children with primary immunodeficiencies with very good results.
An observational, descriptive, longitudinal and retrospective study is carried out. During the years 2008-2019, we evaluated all paediatric patients undergoing HSCT in our center with persistent chronic hypogammaglobulinemia (for over a year). The treatment phase with intravenous immunoglobulin (Privigen®) and the first four years of treatment with subcutaneous immunoglobulin (Hizentra®) are evaluated using a questionnaire.
During the years 2008-2019, 175 patients underwent HSCT, 143 (82%) of whom exceeded three months after transplantation. Three (2%) of them had persistent hypogammaglobulinemia. All three share factors described in the literature involved in immune reconstitution. After analysing the questionnaire, it is observed that switching from intravenous to subcutaneous gammaglobulin has involved a great improvement in their quality of life.
The origin of chronic hypogammaglobulinemia in our patients shows different factors and cannot be attributed to a single cause. Due to the limited number of patients no conclusions can be drawn at the population level. We have been able to observe that replacement treatment with Hizentra 20% has been as effective as the intravenous administration without evidence of an increase in bacterial infections. Furthermore, it has also led to an improvement in quality of life and increased comfort, as the patients themselves have stated.
异基因造血干细胞移植（HSCT）后头几个月患儿常出现低丙种球蛋白血症。在此阶段，必须通过肠外途径给予人免疫球蛋白以预防感染。在某些情况下，这种低丙种球蛋白血症会持续存在，这就迫使患者在通常不再携带中央静脉导管时进行进一步治疗，这使得他们成为皮下替代疗法的理想人选。虽然这种方法在接受 HSCT 的儿科患者中的应用在文献中鲜有报道，但在原发性免疫缺陷儿童的替代治疗中已有广泛描述，且效果非常好。
进行了一项观察性、描述性、纵向和回顾性研究。在 2008 年至 2019 年间，我们评估了在我们中心接受 HSCT 的所有持续性慢性低丙种球蛋白血症（持续一年以上）的儿科患者。使用问卷调查评估静脉注射免疫球蛋白（Privigen®）治疗阶段和皮下免疫球蛋白（Hizentra®）治疗的前四年。
在 2008 年至 2019 年间，有 175 名患者接受了 HSCT，其中 143 名（82%）在移植后超过三个月。其中有 3 名（2%）患者持续性低丙种球蛋白血症。这三人都有文献中描述的涉及免疫重建的因素。分析问卷后，我们发现从静脉注射免疫球蛋白转换为皮下免疫球蛋白极大地提高了他们的生活质量。
我们患者慢性低丙种球蛋白血症的起源显示出不同的因素，不能归因于单一原因。由于患者数量有限，因此不能在人群层面得出结论。我们观察到，20%的 Hizentra 替代治疗与静脉注射一样有效，没有证据表明细菌感染增加。此外，正如患者自己所陈述的，这也提高了生活质量和舒适度。
