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Klin Padiatr 2013; 225(03): 127-132
DOI: 10.1055/s-0033-1333754
Original Article
© Georg Thieme Verlag KG Stuttgart · New York

Normalized Transcranial Doppler Velocities, Stroke Prevention and Improved Pulmonary Function after Stem Cell Transplantation in Children with Sickle Cell Anemia

Normalisierung intrazerebraler Blutflussgeschwindigkeiten, Schlaganfallprophylaxe und verbesserte Lungenfunktion durch Stammzelltransplantation bei Kindern mit Sichelzellanämie
M. Mynarek
1   Kinderheilkunde, Pädiatrische Hämatologie u. Onkologie, Medizinische Hochschule Hannover, Germany
,
C. Bettoni da Cunha Riehm
1   Kinderheilkunde, Pädiatrische Hämatologie u. Onkologie, Medizinische Hochschule Hannover, Germany
,
F. Brinkmann
2   Pädiatrische Pneumologie, Allergologie, Immunologie und Neonatologie, Medizinische Hochschule Hannover, Germany
,
K. Weißenborn
3   Klinik für Neurologie, Medizinische Hochschule Hannover, ­Germany
,
M. Tell-Lüersen
4   Institut für Transfusionsmedizin, Medizinische Hochschule ­Hannover, Germany
,
H.-G. Heuft
4   Institut für Transfusionsmedizin, Medizinische Hochschule ­Hannover, Germany
,
B. Maecker-Kolhoff
1   Kinderheilkunde, Pädiatrische Hämatologie u. Onkologie, Medizinische Hochschule Hannover, Germany
,
K.-W. Sykora
1   Kinderheilkunde, Pädiatrische Hämatologie u. Onkologie, Medizinische Hochschule Hannover, Germany
› Author Affiliations
Further Information

Publication History

Publication Date:
26 April 2013 (online)

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Abstract

Background:

Abnormal transcranial Doppler velocities (TCD) indicate an increased risk of stroke in patients with sickle cell anemia (SCA) and require regular blood transfusions. Hematopoietic stem cell transplantation (HSCT) is under discussion as an alternative to chronic transfusion in these patients.

Patients and methods:

This retrospective analysis includes 9 patients with SCA undergoing HSCT at a single center in Germany. Special focus was given to the neurologic follow-up and to the results of TCD studies.

Results:

High risk of stroke or previous stroke was an HSCT-indication in 8 of 9 patients, although most patients had more than one indication for HSCT. TCD was normalized in all 5 patients after HSCT in whom this test was available. None of the patients developed a stroke after HSCT. No further strokes occurred even in patients that experienced recurrent strokes during chronic transfusion before HSCT. 2 of the 9 patients received a 10/10 HLA-matched unrelated donor graft, the others matched related grafts.

All patients were alive, free of SCA symptoms and transfusion-independent with stable chimerism 3–11 years after HSCT. Pulmonary function tests normalized in 1 patient with severe sickle cell lung disease.

Conclusion:

HSCT is able to prevent stroke in patients with SCA. Its perspectives and limitations should be discussed early during the treatment of a patient with complicated SCA.

Zusammenfassung

Hintergrund:

Erhöhte intrazerebrale Blutflussgeschwindigkeiten (IZF) prädisponieren bei Patienten mit Sichelzellanämie zu Schlaganfällen, und entsprechende Patienten werden routinemäßig mit wiederholten (Austausch-)Transfusionen behandelt. Die hämatopoetische Stammzelltransplantation (SZT) könnte eine therapeutische Alternative für diese Patienten sein.

Patienten und Methoden:

Diese retrospektive Analyse beinhaltet 9 Patienten mit Sichel­zellanämie, die an einem deutschen Zentrum eine hämatopoetische Stammzelltransplantation er­halten haben. Ein Schwerpunkt der Analyse lag auf dem neurologischen Verlauf und der Analyse der Ergebnisse der IZF.

Ergebnisse:

Ein erhöhtes Schlaganfallrisiko war die Indikation zur SZT bei 8 von 9 Patienten, obwohl die meisten mehr als eine Indikation zur SZT hatten. Bei 5 Patienten, bei denen erhöhte IZF vor der SZT bekannt waren, normalisierten sich diese nach der SZT. Kein Patient entwickelte einen Schlaganfall nach SZT, insbesondere auch dann nicht, wenn es bereits vor SZT trotz suffizienter Transfusions­therapie zu rezidivierenden Schlaganfällen gekommen war. 2 Patienten erhielten ein Stammzellpräparat von 10/10 HLA-identischen, unverwandten Spen­dern, die übrigen von HLA-identischen, verwand­ten Spendern.

3–11 Jahre nach Transplantation sind alle Patienten am Leben, transfusionsunabhängig und symptomfrei. Bei einem Patienten mit deutlich reduzierter Lungenfunktion normalisierte sich diese nach SZT.

Schlussfolgerung:

Die SZT kann Schlaganfälle bei Patienten mit Sichelzellanämie verhindern. Deshalb sollten ihre Möglichkeiten und Einschränkungen mit Eltern und Patienten früh diskutiert werden, wenn sich ein komplizierter Verlauf abzeichnet.

Key words

sickle cell anemia - stem cell transplantation - stroke - transcranial doppler

Schlüsselwörter

Sichelzellanämie - Stammzelltransplantation - Schlaganfall - transkranieller Doppler

Supplementary material

 

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