Download What patients to include

Retrospective study: Indication and outcome of hematopoietic stem
cell transplantation in primary immunodeficiency with predominant
hypogammaglobulinemia
Please send the form back either per e-mail, mail or fax.
Dr. Claudia Wehr
[email protected]
or
Dr. Marta Rizzi
[email protected]
or
Prof. Dr. Klaus Warnatz
[email protected]
Centre for Chronic Immunodeficiency
University Medical Centre Freiburg
Breisacher Straße 117 – 2nd floor
79106 Freiburg
Germany
Tel: +49 (0)761 270-77640
Fax: +49 (0)761 270-77600
Have you entered patient data onto the ESID or EBMT data base? □ yes
□ no
If yes, data retrieval is covered by the patient’s respective consent form.
If you have not entered data onto one of the registries, participation to the study
depends on local ethics committee regulations, please contact Dr. Claudia Wehr or
Dr. Marta Rizzi.
The form was completed by:
Name:
Address:
Tel:
Fax:
E-mail:
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I. Patient identification:
Patient identifier (please number your patients, e.g. 001, 002…): ______________________
Date of birth (Note, inclusion criteria: age at transplantation ≥ 2 years): _________________
Sex: ______________________________________________________________________
Ethnicity: __________________________________________________________________
II. Patient characteristics pre-transplant:
a. Clinical data:
PID:______________________________________________________________________
Year of diagnosis: ___________________________________________________________
Year of onset of first symptoms: ________________________________________________
Family history of susceptibility to infections
□ yes
□ no
□ unknown
b. Susceptibility to infections
□ yes
□ no
□ unknown
If yes, please specify predominant type(s) of infection:
__________________________________________________________________________
Warts?
□ yes
□ no
□ unknown
Opportunistic infections?
□ yes
□ no
□ unknown
Chronic viral infections?
□ yes
□ no
□ unknown
If yes, please specify:
__________________________________________________________________________
Did the patient have any type of CNS infection?
□ yes
□ no
□ unknown
If yes, please specify:
__________________________________________________________________________
Major infections (requiring hospitalisation or i.v. anti-infective drugs) in the last year before
HSCT. Please give number and specify type of infection:
__________________________________________________________________________
__________________________________________________________________________
c. PID related complications before HSCT:
Lymphadenopathy?
□ yes, present at HSCT
□ yes, resolved before HSCT
Is there a histology available?
□ yes
□ no
□ no
□ unknown
□ unknown
Please specify site:__________________________________________________________
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Splenomegaly?
□ yes, present at HSCT
□ yes, resolved before HSCT
□ no
□ unknown
□ yes, resolved before HSCT
□ no
□ unknown
Granuloma?
□ yes, present at HSCT
□ yes
Is it histologically proven?
□ no
□ unknown
Please specify site:__________________________________________________________
Lung involvement?
□ yes, present at HSCT
□ yes, resolved before HSCT
□ no
□ unknown
If yes please specify. Interstitial lung disease? Bronchiectasis? Granuloma?
__________________________________________________________________________
Gastrointestinal disease?
□ yes, present at HSCT
□ yes, resolved before HSCT
□ no
□ unknown
If yes, please specify: Chronic diarrhoea? Autoimmune enteropathy? Inflammatory bowel
disease?
__________________________________________________________________________
Autoimmune cytopenia?
□ yes, present at HSCT
□ yes, resolved before HSCT
□ no
□ unknown
If yes, please specify: ITP? Haemolytic anaemia? Autoimmune neutropenia?
__________________________________________________________________________
__________________________________________________________________________
Other forms of autoimmunity?
□ yes, present at HSCT
□ yes, resolved before HSCT
□ no
□ unknown
If yes, please specify:
__________________________________________________________________________
Chronic liver disease?
□ yes, present at HSCT
□ yes, resolved before HSCT
□ no
□ unknown
If yes, please specify:
__________________________________________________________________________
Renal disease?
□ yes, present at HSCT
□ yes, resolved before HSCT
□ no
□ unknown
If yes, please specify:
__________________________________________________________________________
Malignancy?
□ yes, present at HSCT
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□ yes, resolved before HSCT
□ no
□ unknown
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If yes, please specify:
__________________________________________________________________________
Other co-morbidities? Is there something worthwhile to mention about the patient?
__________________________________________________________________________
__________________________________________________________________________
d. Was the patient on immunoglobulin replacement therapy before HSCT?
□ yes
□ no
□ unknown
e. Vaccination response before Ig replacement:
Specific antibody titer to peptide antigen (e.g. tetanus), specify antigen:_________________
□ present
□ not present
□ not available
Specific antibody titer to polysaccharides (e.g. pneumococci), specify antigen:____________
□ present
□ not present
□ not available
f. Bone marrow (BM) aspirate and biopsy pre-transplant. Please specify result or add copy:
________________________________________________________________________
________________________________________________________________________
Plasma cells present in BM?
□ yes
□ no
g. Were immunosuppressive drugs or chemotherapy used before HSCT?
□ yes
□ no
If yes, please specify drug, dose and time:
________________________________________________________________________
________________________________________________________________________
________________________________________________________________________
________________________________________________________________________
h. Is there anything to add in respect to this patient?
________________________________________________________________________
________________________________________________________________________
III. HSCT
a. Indication for HSCT:_______________________________________________________
Date of HSCT:_____________________________________________________________
Karnofsky score (%) before HSCT:_____________________________________________
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b. HLA-match type:
□ HLA-matched family donor
□ HLA-mismatched family donor
□ HLA-matched unrelated donor
□ HLA-mismatched unrelated donor
□ unknown
Donor compatibility:
□ 12/12
Method:
□ 11/12
□ 10/10
□ 9/10
□ antigen matching
Stem cell source:
□ 8/10
□ 6/6
□ 5/6 □ 5/10
□ allelic matching
□ bone marrow
□ cord blood
□ peripheral blood
□ other_________________________
c. Transplant: CD34+ cell count per kg :__________________________________________
d. CMV status of donor
of recipient:
□ positive
□ negative
□ not available
□ positive
□ negative
□ not available
□ yes
□ no
e. Preparative regimen:
Was radiation used during conditioning?
□ unknown
If yes specify dose and time point: _____________________________________________
Conditioning regimen:
Drug: ___________________________________________________________________
Dose:____________________________________________________________________
Drug:____________________________________________________________________
Dose:____________________________________________________________________
Drug:____________________________________________________________________
Dose:____________________________________________________________________
f. GvHD prophylaxis:
Drug:____________________________________________________________________
Dose:____________________________________________________________________
Drug:____________________________________________________________________
Dose:____________________________________________________________________
g. Engraftment:
First day leucocytes 500/µl (date):____________________________________________
First day leucocytes 1.000/µl (date):___________________________________________
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First day thrombocytes  50.000/µl (date):_______________________________________
Lowest haemoglobin documented (value (g/dl) and date):___________________________
Not engrafted □
IV. Follow up:
a. Most recent visit (date): ____________________________________________________
b. Acute GvHD (aGvHD)
□ yes
□ no
□ unknown
Date of diagnosis of aGvHD:_________________________________________________
If yes, specify grade:
□1
Organs involved:
□ skin
□2
□3
□4
□ liver
□ unknown
□ gut
□ unknown
□ other organs, please specify ________________________________________________
Treatment for aGvHD:_______________________________________________________
Start of treatment for aGvHD: ________________________________________________
End of treatment for aGvHD: _________________________________________________
Outcome: □ resolution
□ recurrence
c. Chronic GvHD (cGvHD): □ yes
□ persistence
□ no
□ unknown
□ unknown
Date of diagnosis of cGvHD:__________________________________________________
If yes, specify grade:
□1
Organs involved:
□ skin
□2
□3
□ liver
□4
□ unknown
□ gut
□ unknown
□ other organs, please specify ________________________________________________
Treatment for cGvHD:_______________________________________________________
Start of treatment for cGvHD: ________________________________________________
End of treatment for cGvHD: _________________________________________________
Outcome: □ resolution
□ recurrence
□ persistence
□ unknown
d. Most recent chimerism (date and result):
________________________________________________________________________
In case of incomplete donor chimerism: Is there a line specific chimerism available? Please
specify date and result:
________________________________________________________________________
________________________________________________________________________
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In case of incomplete donor chimerism. Has there at any time been a complete chimerism?
Please specify date: ________________________________________________________
e. Remission from disease for which HSCT was indicated at most recent visit:
□ yes
□ no
□ partial
How was this assessed?
________________________________________________________________________
________________________________________________________________________
Are there any other relevant transplant related complications present at most recent visit?
□ yes
□ no
If yes, please specify:
________________________________________________________________________
________________________________________________________________________
f. Have primary immunodefciency related complications resolved after transplant up to the
most recent visit?
Splenomegaly
□ yes
□ no
□ partial
□ not applicable
Lymphadenopathy
□ yes
□ no
□ partial
□ not applicable
Granuloma
□ yes
□ no
□ partial
□ not applicable
Lung involvement
□ yes
□ no
□ partial
□ not applicable
Gastrointestinal disease
□ yes
□ no
□ partial
□ not applicable
Autoimmune cytopenia
□ yes
□ no
□ partial
□ not applicable
Other forms of autoimmunity
□ yes
□ no
□ partial
□ not applicable
Liver disease
□ yes
□ no
□ partial
□ not applicable
g. Major infections (requiring hospitalisation or i.v. anti-infective drug) after HSCT.
Second year after HSCT (please give number and specify):
________________________________________________________________________
________________________________________________________________________
Last year before most recent visit (please give number and specify):
________________________________________________________________________
________________________________________________________________________
h. Immunoglobulin (Ig) replacement:
Did the patient receive Ig replacement after HSCT?
□ yes
□ no
If no, when was Ig replacement stopped? Please give date: _________________________
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Specific antibody titer to peptide antigen (e.g. tetanus), specify antigen:________________
□ present
□ not present
□ not available
Specific antibody titer to polysaccharides (e.g. pneumococci), specify antigen:__________
□ present
i. Death:
□ not present
□ not available
□ yes
□ no
If yes, please specify date and reason:
________________________________________________________________________
________________________________________________________________________
________________________________________________________________________
V. Time course of immunoglobulin levels:
Ig levels
before Ig
replacement
before
HSCT
12 months
post
HSCT
24 months
post
HSCT
most
recent
Date
IgG (g/l)
IgA (g/l)
IgM (g/l)
VI. Timecourse of lymphocyte counts:
before HSCT
3 months
post HSCT
6 months
post HSCT
12 months
post HSCT
Annually
thereafter
Use extra
sheet if
needed.
Date
absolute
leukocyte
count
(ALC)/µl
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cont.
before HSCT
3 months
post HSCT
6 months
post HSCT
12 months
post HSCT
Annually
thereafter
Use extra
sheet if
needed.
CD3+ /µl
CD4+ /µl
CD8+/µl
CD19 or
CD20+/µl
CD16/
CD56+/µl
VII. Is there any other data on immunological phenotype either pre- or post-transplant
available?
B cell phenotype
□ yes
□ no
□ unknown
T cell activation markers
□ yes
□ no
□ unknown
CD45RA/RO
□ yes
□ no
□ unknown
γ/δ-T cells
□ yes
□ no
□ unknown
small Vβ repertoire
□ yes
□ no
□ unknown
T cell proliferation
□ yes
□ no
□ unknown
If yes please add copy.
Thanks for your effort! We will come back to you with the results of the study.
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