Table 1 Clinical evolution before and after GT

From: Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease

Patient (age at GT time, y)

P1 (19)

P2 (29)

P3 (34)

P4 (15)

Events before GT

Grade 3 VOC (N/y)

14 (0.7)

30 (1)

27 (0.7)

21 (1.3)

ACS (n)

6

2

5

4

Concomitant disorders

(except iron overload)

Osteomyelitis

HBP

PRESS sdr

Guillain Barré sdr

Hearth hypertrophy

Nephropathy

Hypertension

Endocarditis

Osteonecrosis

Nephropathy

Scoliosis

Treatments before GT

ET (period-y)

14

16

5

5

RBC Transfusion Units (N Units per y)

385U (35 U/y)

365U (19.21 U/y)

445 U (22.25 U/y)

125U (9.6U/y)

HU (period-y)

3

13

10

6

Events after GT

Grade 3 VOC (delay of the first episode from GT (M))(N/year)

3 (5.6 m) (1)

2 (8.3 m) (0.6)

1 (2.8 m) (0.5)

3 (2.23 m) (1.5)

Mean hospitalization length (days)

3

4

71

8

ACS (n)

None

None

None

None

SAE (not SCD linked)

Febrile neutropenia°

None

Neuroendocrine tumor

Mucositisa

Treatments after GT

ET (M post-GT)

None

None

2.9b

10.3c

RBC Transfusion Units

(N Units per y)e

None

None

53 U (26.5 U/y)

9 U (4.5U/y)

HU (M post-GT)

7.3d

None

8.9

5

Crizanlizumab

(M post-GT)f

14.7

None

None

None

L-glutamine (M post-GT)

None

None

9.8

None

  1. GT gene therapy, y years, VOC vaso-occlusive crises, ACS acute chest syndrome, HBP high blood pressure, ET exchange transfusion, HU hydroxyurea, M months, sdr syndrome.
  2. aAfter conditioning during aplasia period.
  3. bMonthly; stopped since 1 year.
  4. cBefore potential CVO trigger (Travel to Africa: 2 times).
  5. dP1 received HU from month 7 to month 16 at a dose of 15 mg/kg/d. He restarted HU treatment at month 27 at a dose of 20 mg/kg/d.
  6. eAfter discharged.
  7. fSTOP after 27 months of treatment (EMA recommends revocation of authorization of this treatment in SCD due to poor results of the phase III trial).
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