Table 1 Patient and transplantation characteristics, and transplant outcomes according to ex vivo T-cell depletion methods (N = 115).

From: Improved survival and graft function in ex vivo T-cell depleted haploidentical hematopoietic cell transplantation for primary immunodeficiency

 

CAMPATH-1M

CD34 selection

CD3/CD19 depletion

TCR αβ/CD19 depletion

Number

34

34

7

40

 Year of transplant

1987–1998

1999–2006

2007–2011

2012–2019

Diagnosis

    

 SCID, n (%)

31 (91)

24 (71)

5 (71)

12 (30)

 Non-SCID PID, n (%)

3 (9)

10 (29)

2 (29)

28 (70)

  CD40 ligand deficiency

1

5

0

2

  CGD

0

0

0

4

  MHC class II deficiency

0

0

0

4

  Others

2a

5b

2c

18d

Median age at diagnosis (range), years

 SCID

0.4 (birth—0.9)

0.2 (birth—1.5)

0.5 (0.2–0.7)

0.3 (birth—1.0)

 Non-SCID PID

0.9 (0.4–2.9)

1.1 (0.3–9.8)

0.7 (0.6–0.7)

1.7 (0.1–17.1)

Median age at transplant (range), years

 SCID

0.5 (0.1–1.0)

0.2 (0.1–1.7)

0.8 (0.3–0.9)

0.4 (0.1–1.4)

 Non-SCID PID

2.9 (1.1–5.3)

3.1 (0.5–12.4)

1.1 (0.7–1.5)

2.7 (0.2–18.0)

Median interval between diagnosis and transplant (range), years

 SCID

0.1 (0.03–0.3)

0.1 (0.07–0.2)

0.2 (0.2–0.4)

0.2 (0.02–0.8)

 Non-SCID PID

2.5 (0.2–2.5)

0.7 (0.1–6.6)

0.6 (0.5–0.8)

1.1 (0.1–12.5)

Donor, n (%)

 MUD/MFD

6 (18)

3 (9)

0

0

 MMFD/MMUD (1–2 loci MM)

2 (6)

4 (12)

0

2 (7)

 Haploidentical donor (≥3 loci MM)

26 (76)

27 (79)

7 (100)

38 (95)

  Mother

4

11

6

17

  Father

24

16

1

21

Stem cell source

 Marrow, n (%)

34 (100)

34 (100)

0

0

 PBSC, n (%)

0

0

7 (100)

40 (100)

Graft composition

 Median total nucleated cell dose (range), ×108/kg

5.0 (0.9–11.7)

0.1 (0.02–5.7)

5.5 (1.3–22.6)

10.8 (3.0–96.0)

 Median CD34+ cell dose (range), ×106/kg

3.8 (0.9–18.0)

7.5 (0.2–20.0)

8.4 (2.0–24.0)

17.8 (0.8–60.9)

Conditioning regimen

 None

2 (6)

3 (8)

0

4 (10)

 MAC

11 (32)

21 (62)

0

1 (2)

 RTC

0

0

7 (100)

35 (88)

 RIC

21 (62)

10 (29)

0

0

In vivo serotherapy

 None, n (%)

27 (79)

19 (56)

3 (43)

5 (12)

 ATG, n (%)

3 (9)

2 (6)

4 (57)

33 (83)

 Alemtuzumab, n (%)

4 (12)

13 (38)

0

2 (5)

GVHD prophylaxis

 None, n (%)

31 (91)

7 (21)

1 (14)

21 (53)

 CSA ± MTX/MMF, n (%)

3 (9)

27 (79)

6 (86)

19 (47)

Transplant outcomes

Median duration of follow-up of surviving patients (range), year

21 (5.0–30.5)

16 (5.0–19.3)

7.6 (5.0–9.2)

2.2 (0.6–7.8)

Median day to neutrophil recovery, (range)

27 (14–61)

19 (11–44)

14 (10–19)

14 (7–27)

Acute GvHD, n (%)

 Grade II–IV

8 (23)

2 (6)

0

4 (10)

 Grade III–IV

0

2 (6)

0

1 (3)e

Chronic GvHD, n (%)

0

0

0

0

VOD, n (%)

4 (12)

3 (9)

1 (14)

1 (3)

Number of patients with routine viral surveillance

0

23

7

40

 Any viraemia

NAf

6 (26)

3 (43)

28 (70)

 CMV viraemia

NAf

2 (9)

1 (14)

12 (30)

 Adenoviraemia

NAf

0

1 (14)

16 (40)

 EBV viraemia

NAf

2 (9)

0

16 (40)

 HHV6 viraemia

NAf

2 (9)

1 (14)

7 (18)

Second procedure, n (%)

8 (24)

8 (24)

0

1 (2)

 Stem cell boost

0

7

0

0

 Second HCT

6

1

0

1

 Stem cell boost + second HCT

2

0

0

0

Death, n (%)

14 (41)

11 (32)

2 (29)

6 (15)g

 GvHD

0

1

0

1

 VOD

0

2

0

0

 Infection

12

6

0

1

 Noninfectious respiratory failure

1

2

1

0

 Multi-organ failure

0

0

1

4

 Others

1

0

0

0

  1. ALPS autoimmune lymphoproliferative disease, CID combined immunodeficiency, HLH haemophagocytic lymphohistiocytosis, MAC myeloablative conditioning, MFD matched family donor, MM mismatched, MMFD mismatched family donor, MMUD mismatched unrelated donor, MUD matched unrelated donor, PID primary immunodeficiency, RIC reduced intensity conditioning, RTC reduced toxicity conditioning, SCID severe combined immunodeficiency, WAS Wiskott–Aldrich syndrome.
  2. aHyperIgM syndrome, 1; ZAP70 deficiency, 1.
  3. bCartilage hair hypoplasia, 1; CHARGE syndrome, 1; CID, 1; WAS, 1; ZAP70 deficiency, 1.
  4. cALPS, 1; HLH, 1.
  5. dA20 haploinsufficiency, 1; CID, 1; autoimmune enteropathy, 1; DOCK 8 deficiency, 2; HLH, 1; ICOS, 1; IFGR2, 2; IRF8, 1; PI3 kinase deficiency, 1; reticular dysgenesis, 1; RIPK1, 1; STAT1 deficiency, 1; STAT3 gain of function, 1; TCR2 deficiency, 1; WAS, 2.
  6. eThe patient did not receive in-vivo serotherapy for pre-transplant CMV viraemia and persistent CMV viraemia during HCT.
  7. fNA: surveillance for viral infection was not done during this period.
  8. g5 of 6 deaths had viral infection during transplant.
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