Table 3 Clinical trials in adults

22

Retrospective

Grade IV febrile neutropenia

G-CSF only for neutrophil mobilization; when >10¹⁰ PMNs were infused, clinical benefit compared with historical controls

[89]

11

Case series

Invasive Fusarium infection

Ninety-one percent response rate

[57]

74

Retrospective

Treatment of infections

In 34 patients (46 %), GTXs were discontinued due to clinical response and neutrophil count recovery

[12]

56

Retrospective

Severe infection in SAA

GTX + G-CSF; Survival at 30, 90 and 180 days was 89, 70 and 66 %, respectively. Survival rate correlated with hematopoietic recovery

[58]

24

Retrospective

Invasive opportunistic infections

GTX + IFN-γ1b + G-CSF or GM-CSF. 60 % ORR 4 weeks after treatment

[87]

25

Prospective

Progressive uncontrolled infections

Donors given G-CSF and dexamethasone, either alone or in combination. Favorable responses in 40 % of patients (especially in those with fungal or Gram-negative infections). One death from severe pulmonary reaction

[51]

20

Pilot

Neutropenia refractory to G-CSF

Favorable response in 8 out of 15 assessable patients (53 %)

[53]

19

Phase I/II

Infections after HSCT

GTXs from community donors (94 %). G-CSF + dexamethasone. Resolution of infection in 8/19 patients (42 %). Overall, four of the 19 patients were alive on day 30 after HSCT. None of the patients with invasive aspergillosis (n = 5) cleared the infection

[52]

30

Retrospective

Neutropenia and severe infections

G-CSF + dexamethasone. In 11 patients, resolution of infection could be related to granulocyte transfusions. Three of these patients became long-term survivors

[54]

52

Prospective

Control or prevention of severe infections

Control of infections was achieved in 82 % of life-threatening episodes. No reactivation of infections occurred under prophylactic granulocyte transfusions

[55]

100

Randomized (GRANITE study)

Febrile neutropenia

Ongoing national, multi-center trial; Patients aged 1–75 years (www. drks.de/DRKS00000218); Date of first enrollment: October 2014; Arm 1 (intervention-group): transfusion of standardized leukapheresis products of granulocytes on every other day + standard therapy; arm 2 (control group): standard-therapy without granulocyte transfusions

NA

30

Prospective (GIN1 study)

Febrile neutropenia

Granulocytes derived from whole blood; risk of adverse events comparable to other granulocyte components; recovery of neutrophils and survival in all patients except for two adult patients who died

[26]

114

Randomized (RING study)

Febrile neutropenia

Composite endpoint was survival + microbial response 42 days after randomization; 42 and 43 % success rates for the granulocyte and control groups, respectively

NCT00627393; [56]