J Hematol

Journal of Hematology, ISSN 1927-1212 print, 1927-1220 online, Open Access

Article copyright, the authors; Journal compilation copyright, J Hematol and Elmer Press Inc

Journal website https://jh.elmerpub.com

Original Article

Volume 15, Number 2, April 2026, pages 71-79

Post-Transplant Cyclophosphamide Allows Allogeneic Hematopoietic Stem-Cell Transplantation Across Donor Types for Nonmalignant Hematologic Diseases

↓ Figure 1. Overall survival of the two cohorts: post-transplant cyclophosphamide (PTCY) versus calcineurin inhibitor and methotrexate (CNI-MTX).

↓ Figure 2. Disease-free survival of the two cohorts: post-transplant cyclophosphamide (PTCY) versus calcineurin inhibitor and methotrexate (CNI-MTX).

↓ Figure 3. Graft-versus-host disease-free, relapse-free survival (GRFS) of the two cohorts: post-transplant cyclophosphamide (PTCY) versus calcineurin inhibitor and methotrexate (CNI-MTX).

↓ **Table 1.** Characteristics of Bone Marrow Failure Patients Who Received an Allogeneic Hematopoietic Cell Transplant
Characteristics	PTCY (N = 5)	CNI-MTX (N = 14)	P value
ATG: rabbit anti-thymocyte globulin; CNI: calcineurin inhibitor; MTX: methotrexate; PTCY: post-transplant cyclophosphamide; TBI: total body irradiation.
Age, years, mean (minimum–maximum)	33.8 (21–48)	29.1 (18–58)	0.459
Gender, N (%)			0.1409
Female	1 (20)	9 (64.29)
Male	4 (80)	5 (35.71)
Diagnosis, N (%)			0.4527
Severe aplastic anemia	4 (80)	14 (100)
Diamond-Blackfan anemia	1 (20)	0 (0)
Karnofsky performance status, N (%)			1
90% or more	5 (100)	14 (100)
Donor type, N (%)			0.1838
Sibling donor	1 (20)	6 (42.86)
Matched unrelated donor	2 (40)	7 (50)
Haploidentical	1 (20)	0 (0)
Identical twin donor (syngeneic)	1 (20)	0 (0)
Mismatched unrelated donor (7/8)	0 (0)	1 (7.14)
Graft source, N (%)			0.0844
Bone marrow	2 (40)	12 (85.71)
Peripheral blood stem cells	3 (60)	2 (14.29)
Chimerism, N (%)			0.2112
Full donor	2 (40)	4 (28.57)
Mixed T-cell chimerism; full donor myeloid chimerism	2 (40)	4 (28.57)
None (primary graft failure)	0 (0)	6 (42.86)
Syngeneic donor	1 (20)	0
Conditioning, N (%)			0.2723
Cyclophosphamide and rabbit ATG	0 (0)	3 (21.43)
Fludarabine, cyclophosphamide, rabbit ATG, TBI	5 (100)	11 (78.57)

↓ Table 1. Characteristics of Bone Marrow Failure Patients Who Received an Allogeneic Hematopoietic Cell Transplant

Characteristics

PTCY (N = 5)

CNI-MTX (N = 14)

P value

ATG: rabbit anti-thymocyte globulin; CNI: calcineurin inhibitor; MTX: methotrexate; PTCY: post-transplant cyclophosphamide; TBI: total body irradiation.

Age, years, mean (minimum–maximum)

33.8 (21–48)

29.1 (18–58)

0.459

Gender, N (%)

0.1409

Female

1 (20)

9 (64.29)

Male

4 (80)

5 (35.71)

Diagnosis, N (%)

0.4527

Severe aplastic anemia

4 (80)

14 (100)

Diamond-Blackfan anemia

1 (20)

0 (0)

Karnofsky performance status, N (%)

90% or more

5 (100)

14 (100)

Donor type, N (%)

0.1838

Sibling donor

1 (20)

6 (42.86)

Matched unrelated donor

2 (40)

7 (50)

Haploidentical

1 (20)

0 (0)

Identical twin donor (syngeneic)

1 (20)

0 (0)

Mismatched unrelated donor (7/8)

0 (0)

1 (7.14)

Graft source, N (%)

0.0844

Bone marrow

2 (40)

12 (85.71)

Peripheral blood stem cells

3 (60)

2 (14.29)

Chimerism, N (%)

0.2112

Full donor

2 (40)

4 (28.57)

Mixed T-cell chimerism; full donor myeloid chimerism

2 (40)

4 (28.57)

None (primary graft failure)

0 (0)

6 (42.86)

Syngeneic donor

1 (20)

Conditioning, N (%)

0.2723

Cyclophosphamide and rabbit ATG

0 (0)

3 (21.43)

Fludarabine, cyclophosphamide, rabbit ATG, TBI

5 (100)

11 (78.57)

↓ **Table 2.** Characteristics of Bone Marrow Failure Patients Who Received Allogeneic Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide-Based Conditioning
Age/gender/diagnosis	Prior therapy	Diagnosis to transplant (days)	CD34⁺/kg infused	Donor/graft source	Neutrophil engraftment (days)	Platelet engraftment (days)	Acute GVHD	Chronic GVHD	Chimerism at day 100 Myeloid/T-cell	Infection by day 100	F/U, months
ATG: horse anti-thymocyte globulin; BM: bone marrow; CSA: cyclosporine; DBA: Diamond-Blackfan anemia; GVHD: graft-versus-host disease; MRD: matched related donor; MUD: matched unrelated donor; PBSCs: peripheral blood stem cells; SAA: severe aplastic anemia; F/U: follow-up.
22 years Male SAA	None	105	5.96	Syngeneic PBSC	14	18	No	No	N/A	No	13.6
35 years Male DBA	Prednisone	12,775	5.94	MUD PBSC	14	14	No	No	100%/100%	No	15
48 years Male SAA	None	118	5.96	MRD PBSC	17	31	No	No	100%/93%	No	15.4
43 Male SAA	None	143	2.16	MUD BM	13	18	No	No	100%/81%	No	17.8
21 years Female SAA	ATG, CSA, Eltrombopag	814	2.59	Haploidentical related BM	12	18	No	No	100%/100%	No	18.7

↓ Table 2. Characteristics of Bone Marrow Failure Patients Who Received Allogeneic Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide-Based Conditioning

Age/gender/diagnosis

Prior therapy

Diagnosis to transplant (days)

CD34⁺/kg infused

Donor/graft source

Neutrophil engraftment (days)

Platelet engraftment (days)

Acute GVHD

Chronic GVHD

Chimerism at day 100
Myeloid/T-cell

Infection by day 100

F/U, months

ATG: horse anti-thymocyte globulin; BM: bone marrow; CSA: cyclosporine; DBA: Diamond-Blackfan anemia; GVHD: graft-versus-host disease; MRD: matched related donor; MUD: matched unrelated donor; PBSCs: peripheral blood stem cells; SAA: severe aplastic anemia; F/U: follow-up.

22 years Male
SAA

None

105

5.96

Syngeneic
PBSC

N/A

13.6

35 years Male
DBA

Prednisone

12,775

5.94

MUD
PBSC

100%/100%

48 years Male
SAA

None

118

5.96

MRD
PBSC

100%/93%

15.4

43 Male
SAA

None

143

2.16

MUD
BM

100%/81%

17.8

21 years Female
SAA

ATG, CSA, Eltrombopag

814

2.59

Haploidentical related BM

100%/100%

18.7