Tamburro Robert F, Thomas Neal J, Pon Steven, Jacobs Brian R, Dicarlo Joseph V, Markovitz Barry P, Jefferson Larry S, Willson Douglas F
Department of Pediatrics, Division of Pediatric Critical Care Medicine, Penn State Children's Hospital, The Pennsylvania State University College of Medicine, Hershey, PA, USA. rtamburropsu.edu
Pediatr Crit Care Med. 2008 Sep;9(5):459-64. doi: 10.1097/PCC.0b013e3181849bec.
To assess the impact of calfactant (a modified natural bovine lung surfactant) in immunocompromised children with acute lung injury and to determine the number of patients required for a definitive clinical trial of calfactant in this population.
Post hoc analysis of data from a previous randomized, control trial.
Tertiary care pediatric intensive care units.
All children, defined as immunocompromised, enrolled in a multicenter, masked, randomized, control trial of calfactant for acute lung injury conducted between July 2000 and July 2003.
Patients received either an intratracheal instillation of calfactant or an equal volume of air placebo in a protocolized manner.
Eleven of 22 (50%) calfactant-treated patients died when compared with 18 of 30 (60%) placebo patients (absolute risk reduction 10.0%, 95% confidence interval [CI] -17.3, 37.3). Among the 23 patients with an initial oxygen index (OI) >/=13 and </=37, 44% (4 of 9) of calfactant-treated patients died in comparison with 71% (10 of 14) of placebo (absolute risk reduction 27.0%, 95% CI -13.2, 67.2). Only 33% (3 of 9) of calfactant patients died before intensive care discharge in comparison with 71% (10 of 14) of placebo (absolute risk reduction 38.1%, 95% CI -0.7, 76.9). Calfactant therapy was associated with improved oxygenation in these 23 patients. Using an OI entry criterion of (13 </= OI </= 37), stratifying on the presence of hematopoietic stem cell transplantation, and accepting the 27% difference in mortality observed in this analysis, 63 patients would be required in each arm of a randomized, control trial to demonstrate a significant effect of calfactant on mortality in this patient population assuming a two-sided alpha of 0.05 and a power of 0.85.
These preliminary data suggest a potential benefit of calfactant in this high-risk population. A clinical trial powered to appropriately assess these findings seems warranted and feasible.
评估考福苏（一种改良的天然牛肺表面活性剂）对免疫功能低下的急性肺损伤患儿的影响，并确定在此人群中进行考福苏确定性临床试验所需的患者数量。
对先前一项随机对照试验的数据进行事后分析。
三级医疗儿科重症监护病房。
所有被定义为免疫功能低下的儿童，他们参加了2000年7月至2003年7月期间进行的一项关于考福苏治疗急性肺损伤的多中心、盲法、随机对照试验。
患者按照既定方案接受气管内滴注考福苏或等体积的空气安慰剂。
22名接受考福苏治疗的患者中有11名（50%）死亡，而30名接受安慰剂治疗的患者中有18名（60%）死亡（绝对风险降低10.0%，95%置信区间[CI] -17.3, 37.3）。在初始氧合指数（OI）≥13且≤37的23名患者中，接受考福苏治疗的患者中有44%（9名中的4名）死亡，而接受安慰剂治疗的患者中有71%（14名中的10名）死亡（绝对风险降低27.0%，95% CI -13.2, 67.2）。在重症监护病房出院前，接受考福苏治疗的患者中只有33%（9名中的3名）死亡，而接受安慰剂治疗的患者中有71%（14名中的10名）死亡（绝对风险降低38.1%，95% CI -0.7, 76.9）。考福苏治疗与这23名患者的氧合改善有关。使用OI纳入标准（13≤OI≤37），根据造血干细胞移植的情况进行分层，并接受本分析中观察到的27%的死亡率差异，假设双侧α为0.05且检验效能为0.85，随机对照试验的每组需要63名患者才能证明考福苏对该患者群体的死亡率有显著影响。
这些初步数据表明考福苏在这一高危人群中可能有益。开展一项有足够能力适当评估这些发现的临床试验似乎是必要且可行的。
