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Children’s Memorial Health Institute’s experience in management of PID patients qualified for haematological stem cell transplantation. Beata Wolska – Kuśnierz Department of Immunology CMHI, Warsaw, Poland. Prague May 2006. Primary Immunodeficiencies in CMHI registry 1980 – 200 6 - PowerPoint PPT Presentation
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Children’s Memorial Health Children’s Memorial Health Institute’s experience in Institute’s experience in
management of PID patients management of PID patients qualified for haematological stem qualified for haematological stem
cell transplantationcell transplantation
Beata Wolska – KuśnierzBeata Wolska – KuśnierzDepartment of ImmunologyDepartment of Immunology
CMHI, Warsaw, PolandCMHI, Warsaw, Poland
Prague May 2006
Complement deficiencies
2%
Others4%
Humoral deficiencies
58%
T cell and combined
deficiencies26%Phagocytic
deficiencies10%
Primary Immunodeficiencies Primary Immunodeficiencies in CMHI registry in CMHI registry
19801980 –– 20020066n n == 912 912
2005- Polish National Registry within ESID Online Registry
Absolute indications for HSCT:Absolute indications for HSCT:
severe combined immunodeficiencies severe combined immunodeficiencies
death before 2nd year of age in natural course death before 2nd year of age in natural course of PIDof PID
Realtive indications for HSCTRealtive indications for HSCT : :
increasing PID number for HSCT as alternative increasing PID number for HSCT as alternative
treatmenttreatment
individual qualification based on:individual qualification based on:
PID typePID type
clinical courseclinical course
HSCT donor availabilityHSCT donor availability
age of patientage of patient
social and psychological aspectssocial and psychological aspects
Realtive indications for HSCTRealtive indications for HSCT
Wiskott-Aldrich syndromeWiskott-Aldrich syndrome ( trombocytopenia, infections, risk of ( trombocytopenia, infections, risk of
malignancies )malignancies )
Hiper IgM syndromeHiper IgM syndrome ( infections, liver insufficieniency – ( infections, liver insufficieniency –
Cryptosporidial Cryptosporidial infection – cholangitis scleroticans, high risk of infection – cholangitis scleroticans, high risk of malignancy )malignancy )
Chronic granulomatous diseaseChronic granulomatous disease ( infections, chronic pulmonary disease )( infections, chronic pulmonary disease )
Others PID :..... DNA breakage disorders.....?????Others PID :..... DNA breakage disorders.....?????
1968 – HLA discovery1968 – HLA discovery
1968 – first bone marrow 1968 – first bone marrow transplantation transplantation
from sibling donors in SCID from sibling donors in SCID
and WAS patientsand WAS patients
1997 – first haploidentical BMT in SCID 1997 – first haploidentical BMT in SCID
patient in Poland – BMT Unit patient in Poland – BMT Unit
WroclawWroclaw
HSCT in PID’sHSCT in PID’s - 29 patients - 29 patients
65%
SCIDCIDCD4limfHIMWASCGDagran
41%59%
BM- 12
PBPC - 17
28%
24%35%
MRD - 7
haplo- 8
MUD - 14
PID typePID type Number of patients
Severe combined immunodeficiencies Severe combined immunodeficiencies ( SCID )( SCID )
18
Combined immunodeficiency ( CID )Combined immunodeficiency ( CID ) 1
Primary CD4 lymphopenia ( CD4 limf )Primary CD4 lymphopenia ( CD4 limf ) 1
Hiper IgM syndrome ( HIMS)Hiper IgM syndrome ( HIMS) 3
Wiskott – Aldricha syndrome ( WAS )Wiskott – Aldricha syndrome ( WAS ) 4
Chronic granulomatous disease ( CGD)Chronic granulomatous disease ( CGD) 1
Severe agranulocytosisSevere agranulocytosis 1
79%
25% boys - 23
girls - 6
Age of patients on HSCTAge of patients on HSCT
11%
37%
52%
< 2 r.ż. - 20
2 - 5 r.ż. - 7
> 5 r.ż. - 2
age of patients in months
024487296
120
mediana 14 months 3 months12 months
InitialsInitials SexSex Date of birthDate of birth Year of diagnosisYear of diagnosis DiagnosisDiagnosis HSCTHSCT OutcomeOutcome
MLML FF 1985-11-031985-11-03 19861986 SCIDSCID NoNo DiedDied
SESE FF 1986-11-241986-11-24 19871987 SCIDSCID NoNo DiedDied
MKMK FF 1994-11-091994-11-09 19951995 SCIDSCID NoNo DiedDied
PM M 1995-07-18 1996 SCID 1997 Alive
MJMJ MM 1996-04-281996-04-28 19961996 SCIDSCID NoNo DiedDied
EBEB FF 1997-03-071997-03-07 19971997 OSOS NoNo DiedDied
KK KK MM 1997-10-241997-10-24 19981998 SCIDSCID YesYes AliveAlive
SUSU MM 1997-12-251997-12-25 19981998 SCIDSCID YesYes AliveAlive
DSDS MM 1998-09-231998-09-23 19981998 SCIDSCID YesYes AliveAlive
MTMT FF 1998-11-191998-11-19 19981998 OSOS NoNo DiedDied
KKKK FF 1999-03-081999-03-08 19991999 OSOS YesYes DiedDied
MDMD MM 1999-07-041999-07-04 20002000 SCIDSCID NoNo DiedDied
LWLW FF 2000-03-142000-03-14 20002000 SCID SCID YesYes AliveAlive
MKMK MM 2000-11-062000-11-06 20012001 SCID SCID YesYes AliveAlive
DSDS FF 2001-01-252001-01-25 20012001 SCID SCID YesYes DiedDied
SKSK MM 2001-04-012001-04-01 20012001 OSOS YesYes Alive Alive
ASAS MM 2001-04-162001-04-16 20012001 SCIDSCID NoNo DiedDied
KJKJ MM 2002-01-092002-01-09 20022002 SCID SCID YesYes AliveAlive
GSGS FF 2002-01-082002-01-08 20022002 SCID SCID YesYes AliveAlive
FWFW MM 2002-07-262002-07-26 20032003 SCIDSCID YesYes AliveAlive
MSMS MM 2002-12-302002-12-30 20032003 SCID SCID YesYes AliveAlive
SDSD MM 2003-06-072003-06-07 20032003 SCIDSCID YesYes Alive Alive
JPJP FF 2004-09-132004-09-13 20042004 SCIDSCID YesYes Alive Alive
MNMN FF 2004-07-012004-07-01 20042004 OSOS YesYes AliveAlive
DWDW MM 2004-03-032004-03-03 20042004 SCIDSCID NoNo DiedDied
WWWW FF 2004-03-252004-03-25 20042004 SCIDSCID YesYes AliveAlive
GNGN MM 2005-10-212005-10-21 20052005 OSOS YesYes Died Died
27 patients:27 patients:
21 SCID / 6 OS21 SCID / 6 OS
OUTCOMEOUTCOME
9 died before HSCT9 died before HSCT
3 died after HSCT3 died after HSCT
15 alive after HSCT15 alive after HSCT
Causes of Causes of deaths deaths
before HSCT:before HSCT:BCG itis – 3BCG itis – 3
CMV – 3CMV – 3
P.carini - 1P.carini - 1
Aspergillosis - 2Aspergillosis - 2
SCID
Follow up of 18 SCID patients after Follow up of 18 SCID patients after HSCTHSCT
315
follow up > 6months
deaths
0
24
48
72
96
120
mo
nth
s af
ter
HS
CT
Causes of deaths Number of patients
GvHD IVst.GvHD IVst. 11
SepsisSepsis 11
Renal insufficiencyRenal insufficiency 11
median 35,9 monthsmedian 35,9 months
Follow up of patients other than SCID after Follow up of patients other than SCID after HCSTHCST
Combined immunodeficiency Combined immunodeficiency ( CID )( CID )
1 patient1 patient
1 -died 2 weeks afer HSCT liver insufficiency
Primary CD4 lymphopenia ( CD4 Primary CD4 lymphopenia ( CD4 limf )limf )
1 patient1 patient
1 - alive and well
Hiper IgM syndrome ( HIMS)Hiper IgM syndrome ( HIMS)
3 patient3 patient1 - alive and well1 - died 2 weeks after HSCT –GvHD1 - graft rejection, expecting second procedure
Wiskott – Aldricha syndrome Wiskott – Aldricha syndrome ( WAS )( WAS )
4 patient4 patient
2 - full PID correction 1 - partial PID correction, neurological sequelae1- gratf rejection, expecting second transplant
Chronic granulomatous disease Chronic granulomatous disease ( CGD)( CGD)
1 patient1 patient
1- graft rejection
Severe agranulocytosisSevere agranulocytosis
1 patient1 patient1 - died 3 weeks after HSCT – GvHD
Immunological reconstitution Immunological reconstitution in 23 patients in 23 patients
13%
17%
70%full
partial
none
PID PID correctioncorrection
Number Number
of of patientspatients
Donor Donor chimerismchimerism
CCCC MCMC ARAR
fullfull 1616 1010 66
partial partial 44 44
none none 33 33