IN STROKE-FREE CHILDREN WITH SICKLE CELL ANEMIA AND CEREBRAL VASCULOPATHY, THE OUTCOME OF STENOSIS WAS SIGNIFICANTLY BETTER AFTER TRANSPLANTATION THAN ON CHRONIC TRANSFUSION IN A PROSPECTIVE TRIAL
Author(s): ,
Suzanne Verlhac
Affiliations:
Medical Imagery,CHIC Hospital,Créteil,France
,
Catherine Paillard
Affiliations:
Hemato-Pediatrics,Hautepierre Hospital,Strasbourg,France
,
Charlotte Jubert
Affiliations:
Pediatric Hematology,Bordeaux Hospital,Bordeaux,France
,
Marie Petras
Affiliations:
Referral Center for Sickle Cell Disease,Guadeloupe,Pointe-à Pitre,France
,
Valentine Brousse
Affiliations:
Referral Center for Sickle Cell Disease,Necker Hospital,Paris,France
,
Isabelle Thuret
Affiliations:
Pediatric Hematology,La Timone Hospital,Marseille,France
,
Corinne Pondarré
Affiliations:
Referral Center for Sickle Cell Disease,CHIC Hospital,Créteil,France
,
Florence Missud
Affiliations:
Referral Center for Sickle Cell Disease,Debré Hospital,Paris,France
,
Corinne Guitton
Affiliations:
Referral Center for Sickle Cell Disease,Bicêtre Hospital,Kremlin-Bicêtre,France
,
Mariane de Montalembert
Affiliations:
Referral Center for Sickle Cell Disease,Necker Hospital,Paris,France
,
Cécile Arnaud
Affiliations:
Referral Center for Sickle Cell Disease,CHIC Hospital,Créteil,France
,
Annie Kamdem
Affiliations:
Referral Center for Sickle Cell Disease,CHIC Hospital,Créteil,France
Francoise Bernaudin
Affiliations:
Referral Center for Sickle Cell Disease,CHIC Hospital,Créteil,France
EHA Library. Bernaudin F. 06/16/19; 273258; LB2605
Dr. Francoise Bernaudin
Dr. Francoise Bernaudin
Contributions
Abstract

Abstract: LB2605

Type: Oral Presentation

Presentation during EHA24: On Sunday, June 16, 2019 from 12:15 - 12:30

Location: Hall 5

Background

In children with sickle cell anemia (SCA) cerebral macrovasculopathy detected by transcranial Doppler (TCD) and confirmed by MRA exposes to a high risk of stroke prevented by chronic transfusion or stem cell transplantation (SCT). However, the long-term stenosis outcome has not yet been well described particularly in stroke-free SCA-patients

Aims

The aim of the present study was to perform a detailed post-hoc analysis of the stenosis outcome in the stroke-free patients enrolled in the Drepagreffe trial (NCT01340404). The outcome of velocities at 1 and 3 years has been recently reported (Bernaudin et al, JAMA 2019)

Methods

Sixty-seven SCA-children with a history of abnormal-TCD were enrolled in this prospective trial comparing the cerebral vasculopathy outcome on chronic transfusion or after matched-sibling donor (MSD) SCT. Seven of them had a stroke-history and were excluded from the present study. Cerebral and cervical magnetic-resonance angiography (MRA) were systematically performed at enrollment, 1 and 3 years post-enrollment. Stenosis was defined as narrowing ≥25%. The MRA stenosis-scores, ranging from 0 (best outcome) to 32 (worst outcome) were defined as the weighted sums over the 8 assessed cerebral arteries: right and left middle cerebral (MCA), anterior (ACA), internal carotid (ICA) and extracranial internal carotid arteries (eICA), as 0 if no stenosis, 1 if mild stenosis (25–49%), 2 if moderate stenosis (50–74%), 3 if severe stenosis (75–99%), and 4 if occlusion.

Results

Among the 60 stroke-free patients at enrollment, 28 with a MSD were transplanted while 32 without MSD were maintained on chronic transfusion. After at least one year on transfusion, switch to hydroxyurea was proposed in those with normalized velocities and absence of stenosis. At enrollment 14 patients in each group of treatment had stenosis. In the SCT group 10 patients had intracranial stenosis, 5 had extracranial ICA stenosis associated with intracranial stenosis in 1 patient while in the transfusion group, 9 patients had intracranial stenosis and 8 had extracranial ICA stenosis, associated with intracranial stenosis in 3 patients. At 1-year, in the SCT group, 9 patients had still stenosis: 6 intracranial and 3 extracranial whereas in the transfusion group, 10 had still stenosis: 6 patients had intracranial and 7 extracranial ICA stenosis associated with intracranial stenosis in 3 patients. At 3 years, in the SCT group, 5 patients had still stenosis: 3 in intracranial and 2 in extracranial while in the standard-care group, 10 had stenosis, 6 in intracranial, 7 in extracranial associated with intracranial stenosis in 3 patients. Concerning the outcome of the stenosis-score, in the SCT group, no worsening of stenosis was observed and stenosis was improved in 13/14 and stable in one; by contrast, in the standard-care group, worsening of stenosis score was observed in 6 patients on chronic transfusion (p=0.035), Moreover, the difference between stenosis score at 3 years vs 1 year was significantly different between both groups showing a significantly higher improvement of the stenosis score in the SCT group (mean -1.26 in SCT vs -0.06 in the standard care group (p=0.019)

Conclusion

This prospective trial comparing the outcome of stenosis in stroke-free SCA-patients with history of abnormal-TCD shows a significantly better outcome after SCT than on chronic transfusion encouraging to early systematically recommend SCT in those with stenosis and a MSD.

Session topic: 26. Sickle cell disease

Keyword(s): Cerebrovascular disease, Sickle cell anemia, Stem cell transplant, Transfusion

Abstract: LB2605

Type: Oral Presentation

Presentation during EHA24: On Sunday, June 16, 2019 from 12:15 - 12:30

Location: Hall 5

Background

In children with sickle cell anemia (SCA) cerebral macrovasculopathy detected by transcranial Doppler (TCD) and confirmed by MRA exposes to a high risk of stroke prevented by chronic transfusion or stem cell transplantation (SCT). However, the long-term stenosis outcome has not yet been well described particularly in stroke-free SCA-patients

Aims

The aim of the present study was to perform a detailed post-hoc analysis of the stenosis outcome in the stroke-free patients enrolled in the Drepagreffe trial (NCT01340404). The outcome of velocities at 1 and 3 years has been recently reported (Bernaudin et al, JAMA 2019)

Methods

Sixty-seven SCA-children with a history of abnormal-TCD were enrolled in this prospective trial comparing the cerebral vasculopathy outcome on chronic transfusion or after matched-sibling donor (MSD) SCT. Seven of them had a stroke-history and were excluded from the present study. Cerebral and cervical magnetic-resonance angiography (MRA) were systematically performed at enrollment, 1 and 3 years post-enrollment. Stenosis was defined as narrowing ≥25%. The MRA stenosis-scores, ranging from 0 (best outcome) to 32 (worst outcome) were defined as the weighted sums over the 8 assessed cerebral arteries: right and left middle cerebral (MCA), anterior (ACA), internal carotid (ICA) and extracranial internal carotid arteries (eICA), as 0 if no stenosis, 1 if mild stenosis (25–49%), 2 if moderate stenosis (50–74%), 3 if severe stenosis (75–99%), and 4 if occlusion.

Results

Among the 60 stroke-free patients at enrollment, 28 with a MSD were transplanted while 32 without MSD were maintained on chronic transfusion. After at least one year on transfusion, switch to hydroxyurea was proposed in those with normalized velocities and absence of stenosis. At enrollment 14 patients in each group of treatment had stenosis. In the SCT group 10 patients had intracranial stenosis, 5 had extracranial ICA stenosis associated with intracranial stenosis in 1 patient while in the transfusion group, 9 patients had intracranial stenosis and 8 had extracranial ICA stenosis, associated with intracranial stenosis in 3 patients. At 1-year, in the SCT group, 9 patients had still stenosis: 6 intracranial and 3 extracranial whereas in the transfusion group, 10 had still stenosis: 6 patients had intracranial and 7 extracranial ICA stenosis associated with intracranial stenosis in 3 patients. At 3 years, in the SCT group, 5 patients had still stenosis: 3 in intracranial and 2 in extracranial while in the standard-care group, 10 had stenosis, 6 in intracranial, 7 in extracranial associated with intracranial stenosis in 3 patients. Concerning the outcome of the stenosis-score, in the SCT group, no worsening of stenosis was observed and stenosis was improved in 13/14 and stable in one; by contrast, in the standard-care group, worsening of stenosis score was observed in 6 patients on chronic transfusion (p=0.035), Moreover, the difference between stenosis score at 3 years vs 1 year was significantly different between both groups showing a significantly higher improvement of the stenosis score in the SCT group (mean -1.26 in SCT vs -0.06 in the standard care group (p=0.019)

Conclusion

This prospective trial comparing the outcome of stenosis in stroke-free SCA-patients with history of abnormal-TCD shows a significantly better outcome after SCT than on chronic transfusion encouraging to early systematically recommend SCT in those with stenosis and a MSD.

Session topic: 26. Sickle cell disease

Keyword(s): Cerebrovascular disease, Sickle cell anemia, Stem cell transplant, Transfusion

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