Table 3.
Clinical trials in adults
| # of pts | Study type | Indications for GTX | Remarks/outcome | Reference(s) |
|---|---|---|---|---|
| 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] |
Completed and ongoing clinical trials of therapeutic granulocyte transfusions in adults are summarized
HSCT hematopoietic stem cell transplantation, DXM dexamethasone, OS overall survival, ORR overall response rate