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| Article reference | Subjects | Demographics | Treatment | Conclusion |
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| [9] | 1 | 4 y/o male with SCN | HSCT: matched unrelated donor (MUD) | “HSCT is a useful treatment for SCN patients, especially those who are at high risk for leukemic transformation” |
|
| [10] | 136 | 0–43 y/o male and females with SCN | HSCT
(i) 61 HLA matched related donors
(ii) 61 HLA-MUD
(iii) 14 mismatched donors | “3-year overall survival (OS) was 82%, and transplant-related mortality (TRM) was 17%… Cumulative incidence (1 year) of chronic GVHD was 20%” |
|
| [11] | 7 (one subject transplanted twice) | 2.8–28 y/o males and females with SCN | HSCT
(i) 3 HLA matched siblings
(ii) 3 HLA-MUD
(iii) 2 cord blood | “Two of seven (29%) patients died; both had MDS/L… one patient has chronic GVHD 2 years post-transplant” |
|
| [12] | 18 | 0.2–16.7 y/o males and females with SCN | HSCT
(i) 9 HLA matched siblings
(ii) 9 HLA-MUD | “Engraftment occurred at the first HSCT in 12 patients, four patients received a second HSCT for graft failure, and two patients died. The cause of death was renal failure and graft failure at the first and second HSCT, respectively. The cumulative incidence of grade II–IV acute GVHD and TRM at the first transplantation was 11% and 5.6%, respectively” |
|
| [13] | N/a. Review of guidelines and treatments | Males and females with SCN, leukocyte adhesion deficiency, and chronic granulomatous disease | HSCT with matched and medically unrelated donors | “Allogeneic stem cell transplantation and, possibly, gene-replacement therapy are the only curative treatments available” |
|
| [14] | Review of 300 patients on Severe Chronic Neutropenia International Registry (SCNIR) | Males and females with SCN | (i) GCSF
(ii) HSCT | “More than 90% of patients respond to recombinant human (rHu) G-CSF with ANCs that can be maintained at approximately 1.0 × 10(9)/L… Hematopoietic stem cell transplantation (HSCT) is still the only available treatment for patients refractory to rHuG-CSF treatment” |
|
| [15] | 600 patients with CN collected by the SCNIR | Males and females with SCN | (i) GCSF
(ii) HSCT | “In recent analyses the influence of the G-CSF dose required to achieve neutrophil response (ANC > 1,000/microL) in the risk of developing acute myeloid leukemia (AML) has been reported” |
|
| [7] | 101 SCN, 9 of which received HSCT | Males and females with SCN | HSCT
(i) 2 HLA matched related donors
(ii) 7 HLA-MUD | “HSCT is feasible for patients with SCN who do not respond to G-CSF, who have malignant transformation, or who are at a high risk of malignant transformation, even if an HLA-identical sibling donor is not available” |
|
| [16] | 300 patients from SCNIR | Males and females with SCN | (i) GCSF
(ii) HSCT | “Adverse events documented in this group of patients include splenomegaly, thrombocytopenia, osteoporosis and malignant transformation into MDS/leukemia. If and how rHuG-CSF treatment impacts on these adverse events remains unclear since there are no historical controls for comparison. For those patients who are refractory to rHuG-CSF treatment and continue to have severe and often life-threatening bacterial infections, hematopoietic stem cell transplantation (HSCT) is still the only currently available treatment” |
|
| [17] | N/a (review of characteristics, diagnosis, management, and genetic counseling) | Males and females with SCN | GCSF
(i) HSCT | “Treatment with granulocyte colony-stimulating factor (G-CSF) ameliorates symptoms and reduces infections in almost all affected individuals. For affected individuals with a well-matched donor, hematopoietic stem cell transplantation (HSCT) may be the preferred treatment option. HSCT is the only alternative therapy for individuals with congenital neutropenia who are refractory to high-dose G-CSF or who undergo malignant transformation” |
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