Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis

M C Vonk; Z Marjanovic; F H J van den Hoogen; S Zohar; A V M B Schattenberg; W E Fibbe; J Larghero; E Gluckman; F W M B Preijers; A P J van Dijk; J J Bax; P Roblot; P L C M van Riel; J M van Laar; D Farge

doi:10.1136/ard.2007.071464

Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis

M C Vonk, Z Marjanovic, F H J van den Hoogen, S Zohar, A V M B Schattenberg, W E Fibbe, J Larghero, E Gluckman, F W M B Preijers, A P J van Dijk, J J Bax, P Roblot, P L C M van Riel, J M van Laar, D Farge

Research output: Contribution to journal › Article › Academic › peer-review

Abstract

OBJECTIVE: Systemic sclerosis (SSc) is a generalised autoimmune disease, causing morbidity and a reduced life expectancy, especially in patients with rapidly progressive diffuse cutaneous SSc. As no proven treatment exists, autologous haematopoietic stem cell transplantation (HSCT) is employed as a new therapeutic strategy in patients with a poor prognosis. This study reports the effects on survival, skin and major organ function of HSCT in patients with severe diffuse cutaneous SSc.

PATIENTS AND METHODS: A total of 26 patients were evaluated. Peripheral blood stem cells were collected using cyclophosphamide (4 g/m2) and rHu G-CSF (5 to 10 microg/kg/day) and were reinfused after positive CD34+ selection. For conditioning, cyclophosphamide 200 mg/kg was used.

RESULTS: After a median follow-up of 5.3 (1-7.5) years, 81% (n = 21/26) of the patients demonstrated a clinically beneficial response. The Kaplan-Meier estimated survival at 5 years was 96.2% (95% CI 89-100%) and at 7 years 84.8% (95% CI 70.2-100%) and event-free survival, defined as survival without mortality, relapse or progression of SSc, resulting in major organ dysfunction was 64.3% (95% CI 47.9-86%) at 5 years and 57.1% (95% CI 39.3-83%) at 7 years.

CONCLUSION: This study confirms that autologous HSCT in selected patients with severe diffuse cutaneous SSc results in sustained improvement of skin thickening and stabilisation of organ function up to 7 years after transplantation.

Original language	English
Pages (from-to)	98-104
Number of pages	7
Journal	Annals of the Rheumatic Diseases
Volume	67
Issue number	1
DOIs	https://doi.org/10.1136/ard.2007.071464
Publication status	Published - Jan 2008
Externally published	Yes

Keywords

Adolescent
Adult
Aged
Cyclophosphamide
Disease-Free Survival
Female
Follow-Up Studies
Granulocyte Colony-Stimulating Factor
Hematopoietic Stem Cell Mobilization
Hematopoietic Stem Cell Transplantation
Humans
Linear Models
Male
Middle Aged
Morbidity
Myeloablative Agonists
Recombinant Proteins
Scleroderma, Systemic
Survival Rate
Transplantation Conditioning
Transplantation, Autologous
Clinical Trial, Phase I
Clinical Trial, Phase II
Journal Article
Multicenter Study
Research Support, Non-U.S. Gov't

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10.1136/ard.2007.071464

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Vonk, M. C., Marjanovic, Z., van den Hoogen, F. H. J., Zohar, S., Schattenberg, A. V. M. B., Fibbe, W. E., Larghero, J., Gluckman, E., Preijers, F. W. M. B., van Dijk, A. P. J., Bax, J. J., Roblot, P., van Riel, P. L. C. M., van Laar, J. M., & Farge, D. (2008). Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis. Annals of the Rheumatic Diseases, 67(1), 98-104. https://doi.org/10.1136/ard.2007.071464

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title = "Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis",

abstract = "OBJECTIVE: Systemic sclerosis (SSc) is a generalised autoimmune disease, causing morbidity and a reduced life expectancy, especially in patients with rapidly progressive diffuse cutaneous SSc. As no proven treatment exists, autologous haematopoietic stem cell transplantation (HSCT) is employed as a new therapeutic strategy in patients with a poor prognosis. This study reports the effects on survival, skin and major organ function of HSCT in patients with severe diffuse cutaneous SSc.PATIENTS AND METHODS: A total of 26 patients were evaluated. Peripheral blood stem cells were collected using cyclophosphamide (4 g/m2) and rHu G-CSF (5 to 10 microg/kg/day) and were reinfused after positive CD34+ selection. For conditioning, cyclophosphamide 200 mg/kg was used.RESULTS: After a median follow-up of 5.3 (1-7.5) years, 81% (n = 21/26) of the patients demonstrated a clinically beneficial response. The Kaplan-Meier estimated survival at 5 years was 96.2% (95% CI 89-100%) and at 7 years 84.8% (95% CI 70.2-100%) and event-free survival, defined as survival without mortality, relapse or progression of SSc, resulting in major organ dysfunction was 64.3% (95% CI 47.9-86%) at 5 years and 57.1% (95% CI 39.3-83%) at 7 years.CONCLUSION: This study confirms that autologous HSCT in selected patients with severe diffuse cutaneous SSc results in sustained improvement of skin thickening and stabilisation of organ function up to 7 years after transplantation.",

keywords = "Adolescent, Adult, Aged, Cyclophosphamide, Disease-Free Survival, Female, Follow-Up Studies, Granulocyte Colony-Stimulating Factor, Hematopoietic Stem Cell Mobilization, Hematopoietic Stem Cell Transplantation, Humans, Linear Models, Male, Middle Aged, Morbidity, Myeloablative Agonists, Recombinant Proteins, Scleroderma, Systemic, Survival Rate, Transplantation Conditioning, Transplantation, Autologous, Clinical Trial, Phase I, Clinical Trial, Phase II, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't",

author = "Vonk, {M C} and Z Marjanovic and {van den Hoogen}, {F H J} and S Zohar and Schattenberg, {A V M B} and Fibbe, {W E} and J Larghero and E Gluckman and Preijers, {F W M B} and {van Dijk}, {A P J} and Bax, {J J} and P Roblot and {van Riel}, {P L C M} and {van Laar}, {J M} and D Farge",

year = "2008",

month = jan,

doi = "10.1136/ard.2007.071464",

language = "English",

volume = "67",

pages = "98--104",

journal = "Annals of the Rheumatic Diseases",

issn = "0003-4967",

publisher = "BMJ Publishing Group",

number = "1",

}

Vonk, MC, Marjanovic, Z, van den Hoogen, FHJ, Zohar, S, Schattenberg, AVMB, Fibbe, WE, Larghero, J, Gluckman, E, Preijers, FWMB, van Dijk, APJ, Bax, JJ, Roblot, P, van Riel, PLCM, van Laar, JM & Farge, D 2008, 'Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis', Annals of the Rheumatic Diseases, vol. 67, no. 1, pp. 98-104. https://doi.org/10.1136/ard.2007.071464

TY - JOUR

T1 - Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis

AU - Vonk, M C

AU - Marjanovic, Z

AU - van den Hoogen, F H J

AU - Zohar, S

AU - Schattenberg, A V M B

AU - Fibbe, W E

AU - Larghero, J

AU - Gluckman, E

AU - Preijers, F W M B

AU - van Dijk, A P J

AU - Bax, J J

AU - Roblot, P

AU - van Riel, P L C M

AU - van Laar, J M

AU - Farge, D

PY - 2008/1

Y1 - 2008/1

N2 - OBJECTIVE: Systemic sclerosis (SSc) is a generalised autoimmune disease, causing morbidity and a reduced life expectancy, especially in patients with rapidly progressive diffuse cutaneous SSc. As no proven treatment exists, autologous haematopoietic stem cell transplantation (HSCT) is employed as a new therapeutic strategy in patients with a poor prognosis. This study reports the effects on survival, skin and major organ function of HSCT in patients with severe diffuse cutaneous SSc.PATIENTS AND METHODS: A total of 26 patients were evaluated. Peripheral blood stem cells were collected using cyclophosphamide (4 g/m2) and rHu G-CSF (5 to 10 microg/kg/day) and were reinfused after positive CD34+ selection. For conditioning, cyclophosphamide 200 mg/kg was used.RESULTS: After a median follow-up of 5.3 (1-7.5) years, 81% (n = 21/26) of the patients demonstrated a clinically beneficial response. The Kaplan-Meier estimated survival at 5 years was 96.2% (95% CI 89-100%) and at 7 years 84.8% (95% CI 70.2-100%) and event-free survival, defined as survival without mortality, relapse or progression of SSc, resulting in major organ dysfunction was 64.3% (95% CI 47.9-86%) at 5 years and 57.1% (95% CI 39.3-83%) at 7 years.CONCLUSION: This study confirms that autologous HSCT in selected patients with severe diffuse cutaneous SSc results in sustained improvement of skin thickening and stabilisation of organ function up to 7 years after transplantation.

AB - OBJECTIVE: Systemic sclerosis (SSc) is a generalised autoimmune disease, causing morbidity and a reduced life expectancy, especially in patients with rapidly progressive diffuse cutaneous SSc. As no proven treatment exists, autologous haematopoietic stem cell transplantation (HSCT) is employed as a new therapeutic strategy in patients with a poor prognosis. This study reports the effects on survival, skin and major organ function of HSCT in patients with severe diffuse cutaneous SSc.PATIENTS AND METHODS: A total of 26 patients were evaluated. Peripheral blood stem cells were collected using cyclophosphamide (4 g/m2) and rHu G-CSF (5 to 10 microg/kg/day) and were reinfused after positive CD34+ selection. For conditioning, cyclophosphamide 200 mg/kg was used.RESULTS: After a median follow-up of 5.3 (1-7.5) years, 81% (n = 21/26) of the patients demonstrated a clinically beneficial response. The Kaplan-Meier estimated survival at 5 years was 96.2% (95% CI 89-100%) and at 7 years 84.8% (95% CI 70.2-100%) and event-free survival, defined as survival without mortality, relapse or progression of SSc, resulting in major organ dysfunction was 64.3% (95% CI 47.9-86%) at 5 years and 57.1% (95% CI 39.3-83%) at 7 years.CONCLUSION: This study confirms that autologous HSCT in selected patients with severe diffuse cutaneous SSc results in sustained improvement of skin thickening and stabilisation of organ function up to 7 years after transplantation.

KW - Adolescent

KW - Adult

KW - Aged

KW - Cyclophosphamide

KW - Disease-Free Survival

KW - Female

KW - Follow-Up Studies

KW - Granulocyte Colony-Stimulating Factor

KW - Hematopoietic Stem Cell Mobilization

KW - Hematopoietic Stem Cell Transplantation

KW - Humans

KW - Linear Models

KW - Male

KW - Middle Aged

KW - Morbidity

KW - Myeloablative Agonists

KW - Recombinant Proteins

KW - Scleroderma, Systemic

KW - Survival Rate

KW - Transplantation Conditioning

KW - Transplantation, Autologous

KW - Clinical Trial, Phase I

KW - Clinical Trial, Phase II

KW - Journal Article

KW - Multicenter Study

KW - Research Support, Non-U.S. Gov't

U2 - 10.1136/ard.2007.071464

DO - 10.1136/ard.2007.071464

M3 - Article

C2 - 17526554

SN - 0003-4967

VL - 67

SP - 98

EP - 104

JO - Annals of the Rheumatic Diseases

JF - Annals of the Rheumatic Diseases

IS - 1

ER -

Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis

Abstract

Keywords

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