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Table 2 Clinical trials in children

From: Granulocyte transfusions in children and adults with hematological malignancies: benefits and controversies

# of pts Clinical trial Indications for GTX Remarks/outcome Reference(s)
27 Prospective, phase II Severe neutropenia and infections Donor mobilization: 7.5 μg/kg G-CSF; resolution of infection in 92.6 % of patients; 81.5 % OS on day +30; early administration after a median infection period of 6 days [41]
49 Prospective Neutropenia and invasive bacterial or fungal infection Donor mobilization with 5 μg/kg G-CSF + 50 mg PDN. Mixed cohort, including 10 adults; 72 % OS on day +28 and 52 % OS on day +100 [42]
13 Prospective phase I/II Neutropenia and severe infection Donor mobilization with 5–10 μg/kg G-CSF; Collection through the bag method. 69 % OS on day +30 [90]
3 Prospective CGD and invasive aspergillosis Donor’s mobilization with 450 μg G-CSF + 8 mg DXM; one patient died for ARDS, one was lost at follow-up and died 1 year after discharge, one is alive [49]
35 Retrospective Febrile neutropenia or defective granulocyte function Donor mobilization with 480 μg G-CSF + 8 mg DXM; OS 77.1 and 65.7 %, respectively, on day +30 and +60; 82.4 % infection-related OS [40]
32 Retrospective Sepsis and neutropenia Donor mobilization with single-dose lenograstim + DXM 8 mg; 59 % OS (8/32 pts died for the underlying infection and 8/32 pts for non-infectious causes) [39]
16 Retrospective Severe neutropenia and documented bacterial and/or fungal infections in HSCT recipients Donor mobilization with 8 mg DXM after 2007; unstimulated donors before 2007; 50 % OS on day +30 [37]
10 Retrospective High risk febrile neutropenia with/without microbiologically documented severe infection Donor mobilization with 5 μg/kg G-CSF + 8 mg DXM; Clinical response rate 62.9 %, 40 % infection-related mortality, 40 OS  % [91]
13 Retrospective Febrile neutropenia Resolution of the documented infection in 9/12 (75 %) pts; good early survival (12/14 courses of GTX, 86 %); poor long-term survival (5/13 pts, 39 %) [47]
13 Retrospective Severe infections and neutropenia Donor mobilization with G-CSF 300 μg from day -3; complete or partial recovery in 6 and 3 of the 15 courses of GTX (40 and 20 % respectively) [43]
13 Retrospective Granulocyte dysfunction or severe neutropenia and acute life-threatening infections Donor mobilization with 600 μg G-CSF + 8 mg DXM; complete or partial clinical response in 12/13 pts (92 %); 15 % infection-related mortality and 42 % OS [38]
3 Retrospective Secondary prophylaxis of invasive fungal infections during neutropenic episodes Donor mobilization with G-CSF; concomitant combination antifungal therapy; no infection-related mortality [39]
3 Prospective Prophylaxis in HSC recipients with chronic infections Donor mobilization with 480 μg G-CSF + 7.5 mg DXM; after transplant, no flares of the infections (active S. aureus liver abscesses, chronic pulmonary aspergillosis, soft tissue mucormycosis) [50]
20 Prospective Proven fungal or bacterial infection, unresponsive to anti-microbial therapy (n = 16). Poor control of fungal infection prior to allogeneic HSCT (n = 4) In the curative group, infection was controlled in 11 out of 16 children. All patients treated pre-emptively survived the HSCT procedure [24]
10 Prospective CGD with severe infections Resolution of infection in 9 out of 10 patients, despite the fact that 8 patients were alloimmunized and had poor recovery of transfused granulocytes [65]
  1. Completed and ongoing clinical trials of therapeutic granulocyte transfusions in children are summarized
  2. HSCT hematopoietic stem cell transplantation, DXM dexamethasone, OS overall survival, GTX granulocyte transfusions, CGD chronic granulomatous disease